Members of the Immune Deficiency Cellular Therapy Program regularly publish in peer-reviewed journals. You can find a selection of our most recent papers below. (Updated 12/13/2024)
Systemic Treatment with the Janus Kinase Inhibitor Baricitinib in Ocular Chronic Graft-versus-Host Disease
McManus T, Holtzman NG, Zhao A, Cousineau-Krieger C, Vitale S, FitzGibbon EJ, Payne D, Newgen J, Igbinosun C, Im AP, Peer C, Figg WD, Sr., Cowen EW, Mays JW, Pavletic S, Magone MT
Ophthalmol Sci 2025 5(1): 100627
Targeted therapy in acute T-cell leukemia/lymphoma: are we close?
Mina A
Leukemia & Lymphoma 2024 1-3 Dec 1
A contemporary downtrend in chronic GVHD?
El Jurdi N, Pavletic SZ
Blood Advances 2024 8(22): 5875-5877 Nov 26
High symptom burden in female X-linked chronic granulomatous disease carriers
Miranda MA, Tsalatsanis A, Trotter JR, Arnold DE, Squire JD, Kidd S, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Grunebaum E, Sullivan KE, Newburger PE, Dinauer MC, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Morton FB, Leiding JW
Clinical Immunology 2024 268(110364) Nov
Role of allogeneic hematopoietic cell transplantation in VEXAS syndrome
Dias AL, Groarke EM, Hickstein D, Patel B
Annals of Hematology 2024 103(11): 4427-4436 Nov
Patient-reported outcomes in early phase trials for patients with myelodysplastic syndromes
Mendoza T, King AL, Vera E, Mina A, McGraw K, Pavletic S, Armstrong TS
Seminars in Hematology 2024 61(6): 457-464 Oct 30
Intestinal microbiome and myelodysplastic syndromes: Current state of knowledge and perspectives for future
Simunic M, McGraw K, Pavletic SZ, Rashidi A
Seminars in Hematology 2024 61(6): 442-448 Oct 28
Allogeneic hematopoietic cell transplantation in elderly patients with myelodysplastic syndromes: Considerations and challenges
Schulz E, Pavletic SZ, Mina A
Seminars in Hematology 2024 61(6): 420-430 Oct 17
Taming the flame: WAS and IL-1 blockade
Pai SY
Blood 2024 144(16): 1652-1653 Oct 17
Challenges and solutions for cellular therapy development in autoimmune diseases
Volkmann ER, Varga J, Blazar BR, Pavletic SZ
The Lancet Rheumatology 2024 6(11): e740-e743 Sep 24
Hematopoietic Cell Transplantation for DOCK8 Deficiency: Results from a Prospective Clinical Trial
Freeman AF, Gonzalez CE, Yates B, Cole K, Little L, Flannelly E, Steinberg SM, Mo G, Piette N, Hughes TE, Cuellar-Rodriguez J, Gea-Banacloche J, Heller T, Hammoud DA, Holland SM, Kong HH, Young FD, Jing H, Kayaoglu B, Su HC, Pai SY, Hickstein DD, Shah NN
J Allergy Clin Immunol 2024 Sep 2
Human papillomavirus disease in GATA2 deficiency: a genetic predisposition to HPV-associated female anogenital malignancy
Dancy E, Stratton P, Pichard DC, Marciano BE, Cowen EW, McBride AA, Van Doorslaer K, Merideth MA, Salmeri N, Hughes MS, Heller T, Parta M, Hickstein DD, Kong HH, Holland SM, Zerbe CS
Front Immunol 2024 15(1445711) Aug 29
High-dose alemtuzumab and cyclosporine vs tacrolimus, methotrexate, and sirolimus for chronic graft-versus-host disease prevention
Holtzman NG, Curtis LM, Salit RB, Shaffer BC, Pirsl F, Ostojic A, Steinberg SM, Schulz E, Wilder JS, Hughes TE, Rose J, Memon S, Korngold R, Gea-Banacloche JC, Fowler DH, Hakim FT, Gress RE, Bishop MR, Pavletic SZ
Blood Adv 2024 8(16): 4294-4310 Aug 26
Breaking Boundaries: Immunotherapy for Myeloid Malignancies
Gavrilova T, Schulz E, Mina A
Cancers (Basel) 2024 16(16): Aug 6
Infections and antimicrobial prophylaxis in patients with myelodysplastic syndromes
Czech MM, Schulz E, Mina A, Gea-Banacloche J
Semin Hematol 2024 Aug 3
Association of Busulfan Exposure and Outcomes after HCT for Patients with an Inborn Error of Immunity
Bognàr T, Garcia-Rosa M, Lalmohamed A, Güngör T, Hauri-Hohl MM, Prockop SE, Oram LM, Pai SY, Brooks J, Savic RM, Dvorak CC, Long-Boyle JR, Krajinovic M, Bittencourt H, Teyssier AC, Theoret Y, Martinez C, Egberts TCG, Morales E, Slatter MA, Cuvelier GDE, Chiesa R, Wynn R, Coussons M, Cicalese MP, Ansari M, Long SE, Ebens CL, Lust H, Chaudhury S, Nath CE, Shaw PJ, Keogh Sj Mbbs F, van der Stoep E, Bredius RG, Lindemans CA, Boelens JJ, Bartelink IH
Blood Adv 2024 Jul 29
Opportunistic Infections, Mortality Risk, and Prevention Strategies in Patients With Vacuoles, E1 Enzyme, X-Linked, Autoinflammatory, Somatic (VEXAS) Syndrome
Czech M, Cuellar-Rodriguez J, Patel BA, Groarke EM, Cowen EW, Turturice B, Beck DB, Wilson L, Goodspeed W, Darden I, Young NS, Hickstein D, Ombrello A, Hoffman P, Arikan EA, Sinaii N, Hathaway L, Castelo-Soccio L, Fike A, Kastner DB, Grayson PC, Ferrada MA
Open Forum Infect Dis 2024 11(7): ofae405 Jul 29
Oral and gut microbiome alterations in oral chronic graft-versus-host disease: results from Close Assessment and Testing for Chronic GVHD (CATCH study)
Rashidi A, Pidala J, Hamilton BK, Pavletic SZ, Kim K, Zevin A, Mays JW, Lee SJ
Clinical Cancer Research 2024 30(18): 4240-4250 Jul 17
Study protocol: Close Assessment and Testing for Chronic Graft-vs.-Host disease (CATCH)
Pidala J, Carpenter PA, Onstad L, Pavletic SZ, Hamilton BK, Chen GL, Farhadfar N, Hall M, Lee SJ
PLoS One 2024 19(5): e0298026 May 16
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: a PIDTC study
Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguia-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Davila Saldana BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Malech HL, Kang EM, Leiding JW
J Allergy Clin Immunol 2024 153(5): 1423-1431 e1422 May
High-dose Alemtuzumab-Cyclosporine vs Tacrolimus-Methotrexate-Sirolimus for Chronic Graft-versus-Host Disease Prevention
Holtzman NG, Curtis LM, Salit RB, Shaffer BC, Pirsl F, Ostojic A, Steinberg SM, Schulz E, Wilder JS, Hughes TE, Rose J, Memon S, Korngold R, Gea-Banacloche J, Fowler DH, Hakim FT, Gress RE, Bishop MR, Pavletic SZ
Blood Adv 2024 Apr 26
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment
Keller MD, Hanley PJ, Chi YY, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai SY, Davila Saldana BJ, Hanisch B, Quigg TC, Adams RH, Dahlberg A, Chandrakasan S, Hasan H, Malvar J, Jensen-Wachspress MA, Lazarski CA, Sani G, Idso JM, Lang H, Chansky P, McCann CD, Tanna J, Abraham AA, Webb JL, Shibli A, Keating AK, Satwani P, Muranski P, Hall E, Eckrich MJ, Shereck E, Miller H, Mamcarz E, Agarwal R, De Oliveira SN, Vander Lugt MT, Ebens CL, Aquino VM, Bednarski JJ, Chu J, Parikh S, Whangbo J, Lionakis M, Zambidis ET, Gourdine E, Bollard CM, Pulsipher MA
Nat Commun 15(1): 3258 Apr 18
How I reduce and treat posttransplant relapse of MDS
Mina A, Greenberg PL, Deeg HJ
Blood 2024 143(14): 1344-1354 Apr 4
COVID-19 Vaccination in Patients with Inborn Errors of Immunity Reduces Hospitalization and Critical Care Needs Related to COVID-19: a USIDNET Report
McDonnell, J., Cousins, K., Younger, M.E.M., Lane, A., Abolhassani, H., Abraham, R.S., Al-Tamemi, S., Aldave-Becerra, J.C., Al-Faris, E.H., Alfaro-Murillo, A., AlKhater, S.A., Alsaati, N., Doss, A.M.A., Anderson, M., Angarola, E., Ariue, B., Arnold, D.E., Assa'ad, A.H., Aytekin, C., Bank, M., Bergerson, J.R.E., Bleesing, J., Boesing, J., Bouso, C., Brodszki, N., Cabanillas, D., Cady, C., Callahan, M.A., Caorsi, R., Carbone, J., Carrabba, M., Castagnoli, R., Catanzaro, J.R., Chan, S., Chandra, S., Chapdelaine, H., Chavoshzadeh, Z., Chong, H.J., Connors, L., Consonni, F., Correa-Jimenez, O., Cunningham-Rundles, C., D'Astous-Gauthier, K., Delmonte, O.M., Demirdag, Y.Y., Deshpande, D.R., Diaz-Cabrera, N.M., Dimitriades, V.R., El-Owaidy, R., ElGhazali, G., Al-Hammadi, S., Fabio, G., Faure, A.S., Feng, J., Fernandez, J.M., Fill, L., Franco, G.R., Frenck, R.W., Fuleihan, R.L., Giardino, G., Galant-Swafford, J., Gambineri, E., Garabedian, E.K., Geerlinks, A.V., Goudouris, E., Grecco, O., Pan-Hammarstrom, Q., Khani, H.H.K., Hammarstrom, L., Hartog, N.L., Heimall, J., Hernandez-Molina, G., Horner, C.C., Hostoffer, R.W., Hristova, N., Hsiao, K.C., Ivankovich-Escoto, G., Jaber, F., Jalil, M., Jamee, M., Jean, T., Jeong, S., Jhaveri, D., Jordan, M.B., Joshi, A.Y., Kalkat, A., Kanarek, H.J., Kellner, E.S., Khojah, A., Khoury, R., Kokron, C.M., Kumar, A., Lecerf, K., Lehman, H.K., Leiding, J.W., Lesmana, H., Lim, X.R., Lopes, J.P., Lopez, A.L., Tarquini, L., Lundgren, I.S., Magnusson, J., Marinho, A., Marseglia, G.L., Martone, G.M., Mechtler, A.G., Mendonca, L., Milner, J.D., Mustillo, P.J., Naderi, A.G., Naviglio, S., Nell, J., Niebur, H.B., Notarangelo, L., Oleastro, M., Ortega-Lopez, M.C., Patel, N.R., Petrovic, G., Pignata, C., Porras, O., Prince, B.T., Puck, J.M., Qamar, N., Rabusin, M., Raje, N., Regairaz, L., Risma, K.A., Ristagno, E.H., Routes, J., Roxo-Junior, P., Salemi, N., Scalchunes, C., Schuval, S.J., Seneviratne, S.L., Shankar, A., Sherkat, R., Shin, J.J., Siddiqi, A., Signa, S., Sobh, A., Lima, F.M.S., Stenehjem, K.K., Tam, J.S., Tang, M., Barros, M.T., Verbsky, J., Vergadi, E., Voelker, D.H., Volpi, S., Wall, L.A., Wang, C., Williams, K.W., Wu, E.Y., Wu, S.S., Zhou, J.J., Cook, A., Sullivan, K.E. and Marsh, R.
J Clin Immunol 2024 44(4):86 Apr
The evolution of preclinical models for myelodysplastic neoplasms
Mina A, Pavletic S, Aplan PD
Leukemia 38(4): 683-691 Apr
Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia
Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Davila Saldana BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E
Clin Immunol 261: 109942 Apr
Secondary bone marrow graft loss after third-party virus-specific T cell infusion: Case report of a rare complication
Keller MD, Schattgen SA, Chandrakasan S, Allen EK, Jensen-Wachspress MA, Lazarski CA, Qayed M, Lang H, Hanley PJ, Tanna J, Pai SY, Parikh S, Berger SI, Gottschalk S, Pulsipher MA, Thomas PG, Bollard CM
Nat Commun 2024 15(1): 2749 Mar 29
CAR19 monitoring by peripheral blood immunophenotyping reveals histology-specific expansion and toxicity
Hamilton MP, Craig E, Gentille Sanchez C, Mina A, Tamaresis J, Kirmani N, Ehlinger Z, Syal S, Good Z, Sworder B, Schroers-Martin J, Lu Y, Muffly L, Negrin RS, Arai S, Lowsky R, Meyer E, Rezvani AR, Shizuru JA, Weng WK, Shiraz P, Sidana S, Bharadwaj S, Smith M, Dahiya S, Sahaf B, Kurtz DM, Mackall CL, Tibshirani R, Alizadeh AA, Frank MJ, Miklos DB
Blood Adv, 2024 2024 8(12): 3314-3326 Mar 18
Identification of Fibroinflammatory and Fibrotic Transcriptomic Subsets of Human Cutaneous Sclerotic Chronic Graft-Versus-Host Disease
Rosenstein RK, Rose JJ, Brooks SR, Tsai WL, Gadina M, Pavletic SZ, Nagao K, Cowen EW
JID Innov 4(2): 100246 Mar
Ruxolitinib, IV Immunoglobulin, and High-Dose Glucocorticoids for Critically Ill Adults With Secondary Hemophagocytic Lymphohistiocytosis: A Single-Center Observational Pilot Study
Scholz L, Posch F, Schulz E, Gornicec M, Wolfler A, Reisinger AC, Reinisch A, Eller P, Eisner F, Kreuzer P, Stradner M, Rosenkranz AR, Krammer F, Schilcher G, Krause R, Hatzl S
Crit Care Explor 6(2): e1046 Feb
Heightened TLR7 signaling primes BCR-activated B cells in chronic graft-versus-host disease for effector functions
Bracken SJ, Suthers AN, DiCioccio RA, Su H, Anand S, Poe JC, Jia W, Visentin J, Basher F, Jordan CZ, McManigle WC, Li Z, Hakim FT, Pavletic SZ, Bhuiya NS, Ho VT, Horwitz ME, Chao NJ, Sarantopoulos S
Blood Adv 2024 8(3): 667-680 Feb 13
Development and psychometric testing of a pediatric chronic graft-versus-host disease symptom scale: protocol for a two-phase, mixed methods study
Mitchell SA, Hunter R, Fry A, Pavletic SZ, Widemann BC, Wiener L
Front Psychol 2024 14:1243005 Jan 8
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study
Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O'Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Davila Saldana BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J
J Allergy Clin Immunol 2024 153(1): 287-296 Jan
Red cell distribution width as a new prognostic biomarker in refractory chronic graft-versus-host disease
Schulz E, Pirsl F, Holtzman NG, Beshensky D, Cowen EW, Mitchell SA, Steinberg SM, Pavletic SZ
Haematologica 2023 109(1): 298-302
W"H-Y" antigen/HLA complexes in chronic GVHD
Holtzman NG, Pavletic SZ
Blood 2023 142(11): 946-948
Chronic GvHD NIH Consensus Project Biology Task Force: evolving path to personalized treatment of chronic GvHD
Buxbaum NP, Socie G, Hill GR, MacDonald KPA, Tkachev V, Teshima T, Lee SJ, Ritz J, Sarantopoulos S, Luznik L, Zeng D, Paczesny S, Martin PJ, Pavletic SZ, Schultz KR, Blazar BR
Blood Adv 2023 7(17): 4886-4902
A novel GATA2 distal enhancer mutation results in MonoMAC syndrome in 2 second cousins
West RR, Bauer TR, Tuschong LM, Embree LJ, Calvo KR, Tillo D, Davis J, Holland SM, Hickstein DD
Blood Adv 2023 7(20): 6351-6363
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome
Labrosse R, Chu JI, Armant MA, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, Coenen-van der Spek J, Miggelbrink A, Fiorini C, Adhikari H, Berry CC, Cantu VA, Fong J, Jaroslavsky J, Karadeniz DF, Li QZ, Reddy S, Roche AM, Zhu C, Whangbo JS, Dansereau C, Mackinnon B, Morris E, Koo SM, London WB, Baris S, Ozen A, Karakoc-Aydiner E, Despotovic JM, Forbes Satter LR, Saitoh A, Aizawa Y, King A, Nguyen MAT, Vu VDU, Snapper SB, Galy A, Notarangelo LD, Bushman FD, Williams DA, Pai SY
Blood 2023 142(15): 1281-1296
Predictors and significance of kidney dysfunction in patients with chronic graft-versus-host disease
Beshensky D, Pirsl F, Holtzman NG, Steinberg SM, Mays JW, Cowen EW, Comis LE, Joe GO, Magone MT, Schulz E, Waldman MA, Pavletic SZ
Bone Marrow Transplant 2023 58(10): 1112-1120
Chronic graft-versus-host disease is characterized by high levels and distinctive tissue-of-origin patterns of cell-free DNA
Pang Y, Andargie TE, Jang MK, Kong H, Park W, Hill T, Redekar N, Fu YP, Parth DA, Holtzman NG, Pavletic SZ, Agbor-Enoh S
iScience 2023 26(11): 108160
Thyroid and Adrenal Dysfunction in Hemoglobinopathies Before and After Allogeneic Hematopoietic Cell Transplant
Mandava M, Lew J, Tisdale JF, Limerick E, Fitzhugh CD, Hsieh MM
J Endocr Soc 2023 7(12): bvad134
Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndromes: The Current Landscape and Future Directions
Sabile J, Pavletic S, Migdady Y
Cancer J 2023 29(3): 179-187
The spectrum of GATA2 deficiency syndrome
Calvo KR, Hickstein DD
Blood 2023 141(13): 1524-1532
Therapy-related Myeloid Neoplasms Following PARP Inhibitors-Letter
Batalini F, Mina LA, Mina A
Clin Cancer Res 2023 29(6): 1157
Mass cytometric analysis of the immune cell landscape after traumatic brain injury elucidates the role of complement and complement receptors in neurologic outcomes
Toutonji A, Krieg C, Borucki DM, Mandava M, Guglietta S, Tomlinson S
Acta Neuropathol Commun 2023 11(1): 92
Single-cell landscape analysis unravels molecular programming of the human B cell compartment in chronic GVHD
Poe JC, Fang J, Zhang D, Lee MR, DiCioccio RA, Su H, Qin X, Zhang JY, Visentin J, Bracken SJ, Ho VT, Wang KS, Rose JJ, Pavletic SZ, Hakim FT, Jia W, Suthers AN, Curry-Chisolm IM, Horwitz ME, Rizzieri DA, McManigle WC, Chao NJ, Cardones AR, Xie J, Owzar K, Sarantopoulos S
JCI Insight 2023 8(11): e16973
Immunopathogenic mechanisms and modulatory approaches to graft-versus-host disease prevention in acute myeloid leukaemia
Pang Y, Holtzman NG
Best Pract Res Clin Haematol 2023 36(2): 101475
Alemtuzumab and CXCL9 levels predict likelihood of sustained engraftment after reduced-intensity conditioning HCT
Geerlinks AV, Scull B, Krupski C, Fleischmann R, Pulsipher MA, Eapen M, Connelly JA, Bollard CM, Pai SY, Duncan CN, Kean LS, Baker KS, Burroughs LM, Andolina JR, Shenoy S, Roehrs P, Hanna R, Talano JA, Schultz KR, Stenger EO, Lin H, Zoref-Lorenz A, McClain KL, Jordan MB, Man TK, Allen CE, Marsh RA
Blood Adv 2023 7(14): 3725-3734
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium
Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ, 2nd, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD
Lancet 2023 402(10396): 129-140
The genomic landscape of rare disorders in the Middle East
El Naofal M, Ramaswamy S, Alsarhan A, Nugud A, Sarfraz F, Janbaz H, Taylor A, Jain R, Halabi N, Yaslam S, Alfalasi R, Shenbagam S, Rabea F, Bitzan M, Yavuz L, Wafadari D, Abulhoul H, Shankar S, Al Maazmi M, Rizk R, Alloub Z, Elbashir H, Babiker MOE, Chencheri N, AlBanna A, Sultan M, El Bitar M, Kherani S, Thalange N, Alshryda S, Di Donato R, Tzivinikos C, Majid I, Freeman AF, Gonzalez C, Khan AO, Hamdan H, Abuhammour W, AlAwadhi M, AlKhayat A, Alsheikh-Ali A, Abou Tayoun AN
Genome Med 2023 15(1): 5
Genomic Landscape of Patients with Germline RUNX1 Variants and Familial Platelet Disorder with Myeloid Malignancy
Yu K, Deuitch N, Merguerian M, Cunningham L, Davis J, Bresciani E, Diemer J, Andrews E, Young A, Donovan F, Sood R, Craft K, Chong S, Chandrasekharappa S, Mullikin J, Liu PP
bioRxiv 2023
Chronic gvhd dictionary-eurograft cost action initiative consensus report
Zelic Kerep A, Olivieri A, Schoemans H, Lawitschka A, Halter J, Pulanic D, Dickinson A, Greinix HT, Pavletic SZ, Schultz KR, Lee SJ, Wolff D
Bone Marrow Transplant 2023 58(1): 68-71
Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation
Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Davila Saldana BJ, Eissa H, Goldman FD, Heimall J, O'Reilly R, Chaudhury S, Kolb EA, Shenoy S, Griffith LM, Pulsipher M, Kohn DB, Notarangelo LD, Pai SY, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Barreiro LB, Decaluwe H
J Allergy Clin Immunol 2023 151(1): 260-271
Durable discontinuation of systemic therapy for chronic graft-versus-host disease: myth or reality?
Pavletic SZ, Schultz KR
Haematologica 2023 108(2): 303-305
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions
Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, Abraham RS, Satter LF, Notarangelo LD, Puck JM
J Allergy Clin Immunol 2023 151(2): 539-546
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions
Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM
J Allergy Clin Immunol 2023 151(2): 547-555 e545
Natural history study of patients with familial platelet disorder with associated myeloid malignancy
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 142(25): 2146-2158
Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD
Leiding JW, Arnold DE, Parikh S, Logan B, Marsh RA, Griffith LM, Wu R, Kidd S, Mallhi K, Chellapandian D, Si Lim SJ, Grunebaum E, Falcone EL, Murguia-Favela L, Grossman D, Prasad VK, Heimall JR, Touzot F, Burroughs LM, Bleesing J, Kapoor N, Dara J, Williams O, Kapadia M, Oshrine BR, Bednarski JJ, Rayes A, Chong H, Cuvelier GDE, Forbes Satter LR, Martinez C, Vander Lugt MT, Yu LC, Chandrakasan S, Joshi A, Prockop SE, Davila Saldana BJ, Aquino V, Broglie LA, Ebens CL, Madden LM, DeSantes K, Milner J, Rangarajan HG, Shah AJ, Gillio AP, Knutsen AP, Miller HK, Moore TB, Graham P, Bauchat A, Bunin NJ, Teira P, Petrovic A, Chandra S, Abdel-Azim H, Dorsey MJ, Birbrayer O, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM
Blood 2023 142(24): 2105-2118
Progress in the field of hematopoietic stem cell-based therapies for inborn errors of immunity
Arnold DE, Pai SY
Curr Opin Pediatr 2023 35(6): 663-670
Metabolic syndrome prevalence and impact on outcomes in patients with chronic graft-versus-host disease
Zhao AT, Pirsl F, Steinberg SM, Holtzman NG, Schulz E, Mina A, Mays JW, Cowen EW, Comis LE, Joe GO, Yanovski JA, Pavletic SZ
Bone Marrow Transplant 2023 58(12): 1377-1383
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease
Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL
J Allergy Clin Immunol 2023 152(6): 1619-1633 e1611
Moving toward a conceptualization of measurable residual disease in myelodysplastic syndromes
Schulz E, Aplan PD, Freeman SD, Pavletic SZ
Blood Adv 2023 7(16): 4381-4394
GATA2 Deficiency: Predisposition to Myeloid Malignancy and Hematopoietic Cell Transplantation
Rajput RV, Arnold DE
Curr Hematol Malig Rep 2023 18(4): 89-97
Forty years of hematopoietic stem cell transplantation in Croatia: First-World options and First-World science
Pavletic SZ
Croat Med J 2023 64(2): 65-66
Differential diagnosis of bone marrow failure syndromes guided by machine learning
Gutierrez-Rodrigues F, Munger E, Ma X, Groarke EM, Tang Y, Patel BA, Catto LFB, Cle DV, Niewisch MR, Alves-Paiva RM, Donaires FS, Pinto AL, Borges G, Santana BA, McReynolds LJ, Giri N, Altintas B, Fan X, Shalhoub R, Siwy CM, Diamond C, Raffo DQ, Craft K, Kajigaya S, Summers RM, Liu P, Cunningham L, Hickstein DD, Dunbar CE, Pasquini R, De Oliveira MM, Velloso E, Alter BP, Savage SA, Bonfim C, Wu CO, Calado RT, Young NS
Blood 2023 141(17): 2100-2113
A conversation with Steven Pavletic, MD, MS, senior clinician, National Cancer Institute, Center for Cancer Research
Siranosian K
J Clin Transl Sci 2023 7(1): e86
Editorial: Controversies and expectations for prevention and treatment of graft-versus-host-disease: a biological and clinical perspective
Mariotti J, Fowler DH, Bramanti S, Pavletic SZ
Front Immunol 2023 14(1212756
A starting point for the phenotypic classification of pulmonary chronic graft-versus-host disease
Pang Y, Charya AV, Keller MB, Sirajuddin A, Fu YP, Holtzman NG, Pavletic SZ, Agbor-Enoh S
Blood Adv 2022 6(17): 4987-4988
Correlation of Patient-Reported Outcomes with Clinical Organ Responses: Data from the Belumosudil Chronic Graft-versus-Host Disease Studies
Lee SJ, Cutler C, Blazar BR, Tu A, Yang Z, Pavletic SZ
Transplant Cell Ther 2022 28(10): 700 e701-700 e706
Collection of peripheral blood mononucleated cells for chronic graft-versus-host disease immunology research: safety and effectiveness of leukapheresis in 132 patients
Mina A, Curtis L, West K, Yau YY, Cowen EW, Hakim F, Pavletic SZ
J Transl Med 2022 20(1): 519
Characterization of Hepatic Dysfunction in Subjects Diagnosed With Chronic GVHD by NIH Consensus Criteria
Yang AH, Han MAT, Samala N, Rizvi BS, Marchalik R, Etzion O, Wright EC, Cao L, Hakim FT, Jones E, Kapuria D, Hickstein DD, Fowler D, Kanakry JA, Kanakry CG, Kleiner DE, Koh C, Pavletic SZ, Heller T
Transplant Cell Ther 2022 28(11): 747 e741-747 e710
Tandem hematopoietic stem cell transplant considerations in families with multiple siblings affected by DOCK8 deficiency
Silbert S, Cole K, Bedoya SZ, Freeman AF, Whangbo JS, Avila DN, Su HC, Yates B, Epstein M, Wendler DS, Pai SY, Hickstein DD, Wiener L, Shah NN
Bone Marrow Transplant 2022 57(11): 1721-1723
Signal Transducer and Activator of Transcription 5B Deficiency-associated Lung Disease
Krone KA, Foley CL, Fishman MP, Vargas SO, Forbes LR, Vece TJ, Al-Herz W, Carey B, Pai SY, Hwa V, Trapnell BC
Am J Respir Crit Care Med 2022 205(10): 1245-1250
Prospective Phase II Trial of Montelukast to Treat Bronchiolitis Obliterans Syndrome after Hematopoietic Cell Transplantation and Investigation into Bronchiolitis Obliterans Syndrome Pathogenesis
Williams KM, Pavletic SZ, Lee SJ, Martin PJ, Farthing DE, Hakim FT, Rose J, Manning-Geist BL, Gea-Banacloche JC, Comis LE, Cowen EW, Justus DG, Baird K, Cheng GS, Avila D, Steinberg SM, Mitchell SA, Gress RE
Transplant Cell Ther 2022 28(5): 264 e261-264 e269
Meaningful response criteria for myelodysplastic syndromes
Kim N, Pavletic S, Norsworthy KJ
Br J Haematol 2022 196(5): 1137-1148
Determinants and Clinical Significance of Musculoskeletal Symptoms in Patients With Chronic Graft-Versus-Host Disease
Zelic Kerep A, Pirsl F, Steinberg S, Mitchell S, Curtis L, Holtzman N, Goklemez S, Bilic E, Cowen E, Pichard D, Joe G, Comis L, Im A, Berger A, Parsons-Wandell L, Pulanic D, Baird K, Gress R, Pavletic S
Hemasphere 2022 6(6): e730
Male-Specific Late Effects in Adult Hematopoietic Cell Transplantation Recipients: A Systematic Review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation
Phelan R, Im A, Hunter RL, Inamoto Y, Lupo-Stanghellini MT, Rovo A, Badawy SM, Burns L, Eissa H, Murthy HS, Prasad P, Sharma A, Suelzer E, Agrawal V, Aljurf M, Baker K, Basak GW, Buchbinder D, DeFilipp Z, Grkovic LD, Dias A, Einsele H, Eisenberg ML, Epperla N, Farhadfar N, Flatau A, Gale RP, Greinix H, Hamilton BK, Hashmi S, Hematti P, Jamani K, Maharaj D, Murray J, Naik S, Nathan S, Pavletic S, Peric Z, Pulanic D, Ross R, Salonia A, Sanchez-Ortega I, Savani BN, Schechter T, Shah AJ, Smith SM, Snowden JA, Steinberg A, Tremblay D, Vij SC, Walker L, Wolff D, Yared JA, Schoemans H, Tichelli A
Transplant Cell Ther 2022 28(6): 335 e331-335 e317
The ISHLT chronic lung allograft dysfunction consensus criteria are applicable to pulmonary chronic graft-versus-host disease
Pang Y, Charya AV, Keller MB, Sirajuddin A, Fu YP, Holtzman NG, Pavletic SZ, Agbor-Enoh S
Blood Adv 2022 6(14): 4196-4207
Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy
Arnold DE, Chellapandian D, Parikh S, Mallhi K, Marsh RA, Heimall JR, Grossman D, Chitty-Lopez M, Murguia-Favela L, Gennery AR, Boulad F, Arbuckle E, Cowan MJ, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Kang EM, Malech HL, Leiding JW
J Clin Immunol 2022 42(5): 1026-1035
Salivary ZG16B expression loss follows exocrine gland dysfunction related to oral chronic graft-versus-host disease
Costa-da-Silva AC, Aure MH, Dodge J, Martin D, Dhamala S, Cho M, Rose JJ, Bassim CW, Ambatipudi K, Hakim FT, Pavletic SZ, Mays JW
iScience 2022 25(1): 103592
Donor source and post-transplantation cyclophosphamide influence outcome in allogeneic stem cell transplantation for GATA2 deficiency
Nichols-Vinueza DX, Parta M, Shah NN, Cuellar-Rodriguez JM, Bauer TR, Jr., West RR, Hsu AP, Calvo KR, Steinberg SM, Notarangelo LD, Holland SM, Hickstein DD
Br J Haematol 2022 196(1): 169-178
Characteristics and outcomes of autoimmune hemolytic anemia after pediatric allogeneic stem cell transplant
Hillier K, Harris EM, Berbert L, Pai SY, Grace RF
Pediatr Blood Cancer 2022 69(1): e29410
CAR T-cell therapy for indolent lymphoma: a new treatment paradigm?
Holtzman NG, Shah NN
Lancet Oncol 2022 23(1): 6-8
ASXL1 and STAG2 are common mutations in GATA2 deficiency patients with bone marrow disease and myelodysplastic syndrome
West RR, Calvo KR, Embree LJ, Wang W, Tuschong LM, Bauer TR, Tillo D, Lack J, Droll S, Hsu AP, Holland SM, Hickstein DD
Blood Adv 2022 6(3): 793-807
Pediatric immune deficiencies: current treatment approaches
Gonzalez C
Curr Opin Pediatr 2022 34(1): 61-70
The clinical landscape of chronic graft-versus-host disease management in 2021
Holtzman NG, Pavletic SZ
Br J Haematol 2022 196(4): 830-848
Clinical response to belumosudil in bronchiolitis obliterans syndrome: a combined analysis from 2 prospective trials
DeFilipp Z, Kim HT, Yang Z, Noonan J, Blazar BR, Lee SJ, Pavletic SZ, Cutler C
Blood Adv 2022 6(24): 6263-6270
Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 1
Smith RH, Bloomer H, Fink D, Keyvanfar K, Nasimuzzaman M, Sancheznieto F, Dutta R, Guenther Bui K, Alvarado LJ, Bauer TR, Jr., Hickstein DD, Russell DW, Malik P, van der Loo JCM, Highfill SL, Kuhns DB, Pirooznia M, Larochelle A
Hum Gene Ther 2022 33(23-24): 1293-1304
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC
Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, Kuo CY, Griffith LM, Liu X, Yip A, Hershfield MS, Ayoub PG, Moore TB, Dorsey MJ, O'Reilly RJ, Kapoor N, Pai SY, Kapadia M, Ebens CL, Forbes Satter LR, Burroughs LM, Petrovic A, Chellapandian D, Heimall J, Shyr DC, Rayes A, Bednarski JJ, Chandra S, Chandrakasan S, Gillio AP, Madden L, Quigg TC, Caywood EH, Davila Saldana BJ, DeSantes K, Eissa H, Goldman FD, Rozmus J, Shah AJ, Vander Lugt MT, Thakar MS, Parrott RE, Martinez C, Leiding JW, Torgerson TR, Pulsipher MA, Notarangelo LD, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Kohn DB
Blood 2022 140(7): 685-705
Long-term follow-up after lymphodepleting autologous haematopoietic cell transplantation for treatment-resistant systemic lupus erythematosus
Goklemez S, Hasni S, Hakim FT, Muraro PA, Pirsl F, Rose J, Memon S, Fowler DF, Steinberg SM, Baker EH, Panch SR, Gress R, Illei GG, Lipsky PE, Pavletic SZ
Rheumatology (Oxford) 2022 61(8): 3317-3328
A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System
Porteus MH, Pavel-Dinu M, Pai SY
Hematol Oncol Clin North Am 2022 36(4): 647-665
Toward a Better Understanding of the Atypical Features of Chronic Graft-Versus-Host Disease: A Report from the 2020 National Institutes of Health Consensus Project Task Force
Cuvelier GDE, Schoettler M, Buxbaum NP, Pinal-Fernandez I, Schmalzing M, Distler JHW, Penack O, Santomasso BD, Zeiser R, Angstwurm K, MacDonald KPA, Kimberly WT, Taylor N, Bilic E, Banas B, Buettner-Herold M, Sinha N, Greinix HT, Pidala J, Schultz KR, Williams KM, Inamoto Y, Cutler C, Griffith LM, Lee SJ, Sarantopoulos S, Pavletic SZ, Wolff D
Transplant Cell Ther 2022 28(8): 426-445
Current Status and Future Directions of Gene-Based Therapy for Pediatric Blood Diseases
Shah NN, Pai SY
Hematol Oncol Clin North Am 2022 36(4): xiii-xiv
Abnormal liver tests are not sufficient for diagnosis of hepatic graft-versus-host disease in critically ill patients
Yang AH, Han MAT, Samala N, Rizvi BS, Marchalik R, Etzion O, Wright EC, Patel R, Khan V, Kapuria D, Samala Venkat V, Kleiner DE, Koh C, Kanakry JA, Kanakry CG, Pavletic S, Williams KM, Heller T
Hepatol Commun 2022 6(8): 2210-2220
miR-181c regulates MCL1 and cell survival in GATA2 deficient cells
Wang W, Chen R, Droll S, Barber E, Saleh L, Corrigan-Cummins M, Trick M, Anastas V, Hawk NV, Zhao Z, Vinh DC, Hsu A, Hickstein DD, Holland SM, Calvo KR
J Leukoc Biol 2022 111(4): 805-816
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2020 Treatment of Chronic GVHD Report
DeFilipp Z, Couriel DR, Lazaryan A, Bhatt VR, Buxbaum NP, Alousi AM, Olivieri A, Pulanic D, Halter JP, Henderson LA, Zeiser R, Gooley TA, MacDonald KPA, Wolff D, Schultz KR, Paczesny S, Inamoto Y, Cutler CS, Kitko CL, Pidala JA, Lee SJ, Socie G, Sarantopoulos S, Pavletic SZ, Martin PJ, Blazar BR, Greinix HT
Transplant Cell Ther 2021 27(9): 729-737
Built to last: gene therapy for ADA SCID
Pai SY
Blood 2021 138(15): 1287-1288
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. The 2020 Highly morbid forms report
Wolff D, Radojcic V, Lafyatis R, Cinar R, Rosenstein RK, Cowen EW, Cheng GS, Sheshadri A, Bergeron A, Williams KM, Todd JL, Teshima T, Cuvelier GDE, Holler E, McCurdy SR, Jenq RR, Hanash AM, Jacobsohn D, Santomasso BD, Jain S, Ogawa Y, Steven P, Luo ZK, Dietrich-Ntoukas T, Saban D, Bilic E, Penack O, Griffith LM, Cowden M, Martin PJ, Greinix HT, Sarantopoulos S, Socie G, Blazar BR, Pidala J, Kitko CL, Couriel DR, Cutler C, Schultz KR, Pavletic SZ, Lee SJ, Paczesny S
Transplant Cell Ther 2021 27(10): 817-835
Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients
Hashem H, Bucciol G, Ozen S, Unal S, Bozkaya IO, Akarsu N, Taskinen M, Koskenvuo M, Saarela J, Dimitrova D, Hickstein DD, Hsu AP, Holland SM, Krance R, Sasa G, Kumar AR, Muller I, de Sousa MA, Delafontaine S, Moens L, Babor F, Barzaghi F, Cicalese MP, Bredius R, van Montfrans J, Baretta V, Cesaro S, Stepensky P, Benedicte N, Moshous D, Le Guenno G, Boutboul D, Dalal J, Brooks JP, Dokmeci E, Dara J, Lucas CL, Hambleton S, Wilson K, Jolles S, Koc Y, Gungor T, Schnider C, Candotti F, Steinmann S, Schulz A, Chambers C, Hershfield M, Ombrello A, Kanakry JA, Meyts I
J Clin Immunol 2021 41(7): 1633-1647
Pulmonary Manifestations of GATA2 Deficiency
Marciano BE, Olivier KN, Folio LR, Zerbe CS, Hsu AP, Freeman AF, Filie AC, Spinner MA, Sanchez LA, Lovell JP, Parta M, Cuellar-Rodriguez JM, Hickstein DD, Holland SM
Chest 2021 160(4): 1350-1359
Anti-plasma cell treatment in refractory autoimmune hemolytic anemia in a child with multivisceral transplant
Ghobrial S, Gonzalez CE, Kaufman S, Yazigi N, Matsumoto C, Fishbein T, Hawksworth J, Ekong UD, Kroemer A, Khan K
Pediatr Transplant 2021 25(7): e14045
Subsequent Cancers in Patients Affected with Moderate or Severe Chronic Graft-versus-Host Disease
Schaar DA, Pirsl F, Holtzman N, Steinberg SM, Nashed J, Ruben C, Cowen EW, Mays JW, Mitchell S, Ostojic A, Munshi PN, Joe GO, Comis LE, Morton L, Pavletic SZ
Transplant Cell Ther 2021 27(11): 937 e931-937 e937
The impact of bridging therapy prior to CD19-directed chimeric antigen receptor T-cell therapy in patients with large B-cell lymphoma
Lutfi F, Holtzman NG, Kansagra AJ, Mustafa Ali M, Bukhari A, Yan J, Samanta S, Gottlieb D, Kim DW, Matsumoto LR, Gahres N, Ruehle K, Lee ST, Law JY, Kocoglu MH, Atanackovic D, Yared JA, Hardy NM, Molitoris J, Mohindra P, Rapoport AP, Dahiya S
Br J Haematol 2021 195(3): 405-412
Efficacy and feasibility of ruxolitinib in chronic steroid-refractory GVHD in a pediatric intestine transplant
Ghobrial S, Gonzalez C, Yazigi N, Kaufman S, Matsumoto C, Fishbein T, Hawksworth J, Kroemer A, Khan K
Pediatr Transplant 2021 25(3): e13836
HPV32-related Heck's disease in a chronic graft-versus-host disease patient with long-term successful KTP laser treatment: A rare case report
Nguyen JT, Allen CT, Dodge JT, Van Doorslaer K, McBride AA, Pavletic SZ, Mays JW
Clin Case Rep 2021 9(5): e04253
Hematopoietic Cell Transplantation and Outcomes Related to Human Papillomavirus Disease in GATA2 Deficiency
Parta M, Cole K, Avila D, Duncan L, Baird K, Schuver BB, Wilder J, Palmer C, Daub J, Hsu AP, Zerbe CS, Marciano BE, Cuellar-Rodriguez JM, Bauer TR, Nason M, Calvo KR, Merideth M, Stratton P, DeCherney A, Shah NN, Holland SM, Hickstein DD
Transplant Cell Ther 2021 27(5): 435 e431-435 e411
Blueprint for the discovery of biomarkers of toxicity and efficacy for CAR T cells and T-cell engagers
Paczesny S, Pasquini MC, Pavletic SZ, Agarwal A, Spellman S, Kean L, Bernatchez C, Gust J, Staedtke V, Perales MA
Blood Adv 2021 5(11): 2519-2522
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2020 Etiology and Prevention Working Group Report
Williams KM, Inamoto Y, Im A, Hamilton B, Koreth J, Arora M, Pusic I, Mays JW, Carpenter PA, Luznik L, Reddy P, Ritz J, Greinix H, Paczesny S, Blazar BR, Pidala J, Cutler C, Wolff D, Schultz KR, Pavletic SZ, Lee SJ, Martin PJ, Socie G, Sarantopoulos S
Transplant Cell Ther 2021 27(6): 452-466
Venous thromboembolism incidence and risk factors in adults with acute lymphoblastic leukemia treated with and without pegylated E. coli asparaginase-containing regimens
Kashanian SM, Holtzman NG, Patzke CL, Cornu J, Duffy A, Koka M, Niyongere S, Duong VH, Baer MR, Apata J, Kamangar F, Emadi A
Cancer Chemother Pharmacol 2021 87(6): 817-826
The Future of Chronic Graft-Versus-Host Disease: Introduction to the 2020 National Institutes of Health Consensus Development Project Reports
Pavletic SZ, Martin PJ, Schultz KR, Lee SJ
Transplant Cell Ther 2021 27(6): 448-451
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-H
Kitko CL, Pidala J, Schoemans HM, Lawitschka A, Flowers ME, Cowen EW, Tkaczyk E, Farhadfar N, Jain S, Steven P, Luo ZK, Ogawa Y, Stern M, Yanik GA, Cuvelier GDE, Cheng GS, Holtan SG, Schultz KR, Martin PJ, Lee SJ, Pavletic SZ, Wolff D, Paczesny S, Blazar BR, Sarantopoulos S, Socie G, Greinix H, Cutler C
Transplant Cell Ther 2021 27(7): 545-557
Predictors of hematologic malignancy relapse in patients with advanced chronic graft-versus-host disease
Ruben CL, Pirsl F, Steinberg SM, Holtzman NG, Parsons-Wandell L, Baruffaldi J, Curtis LM, Mitchell SA, Kerep AZ, Cowen EW, Berger A, Joe GO, Datiles MB, 3rd, Mays JW, Pavletic SZ
Bone Marrow Transplant 2021 56(7): 1584-1592
Immune effector cell-associated neurotoxicity syndrome after chimeric antigen receptor T-cell therapy for lymphoma: predictive biomarkers and clinical outcomes
Holtzman NG, Xie H, Bentzen S, Kesari V, Bukhari A, El Chaer F, Lutfi F, Siglin J, Hutnick E, Gahres N, Ruehle K, Ahmad H, Shanholtz C, Kocoglu MH, Badros AZ, Yared JA, Hardy NM, Rapoport AP, Dahiya S
Neuro Oncol 2021 23(1): 112-121
Glycolytic metabolism of pathogenic T cells enables early detection of GVHD by 13C-MRI
Assmann JC, Farthing DE, Saito K, Maglakelidze N, Oliver B, Warrick KA, Sourbier C, Ricketts CJ, Meyer TJ, Pavletic SZ, Linehan WM, Krishna MC, Gress RE, Buxbaum NP
Blood 2021 137(1): 126-137
Post-transplant multimorbidity index and quality of life in patients with chronic graft-versus-host disease-results from a joint evaluation of a prospective German multicenter validation trial and a cohort from the National Institutes of Health
Wolff D, Herzberg PY, Herrmann A, Pavletic SZ, Heussner P, Mumm F, Hofer C, Hilgendorf I, Hemmati PG, Holler E, Greinix H, Mitchell SA
Bone Marrow Transplant 2021 56(1): 243-256
Hematopoietic Cell Transplantation in the Treatment of Newly Diagnosed Adult Acute Myeloid Leukemia: An Evidence-Based Review from the American Society of Transplantation and Cellular Therapy
Dholaria B, Savani BN, Hamilton BK, Oran B, Liu HD, Tallman MS, Ciurea SO, Holtzman NG, Ii GLP, Devine SM, Mannis G, Grunwald MR, Appelbaum F, Rodriguez C, El Chaer F, Shah N, Hashmi SK, Kharfan-Dabaja MA, DeFilipp Z, Aljurf M, AlShaibani A, Inamoto Y, Jain T, Majhail N, Perales MA, Mohty M, Hamadani M, Carpenter PA, Nagler A
Transplant Cell Ther 2021 27(1): 6-20
A randomized phase 2 trial of pomalidomide in subjects failing prior therapy for chronic graft-versus-host disease
Curtis LM, Ostojic A, Venzon DJ, Holtzman NG, Pirsl F, Kuzmina ZJ, Baird K, Rose JJ, Cowen EW, Mays JW, Mitchell SA, Parsons-Wandell L, Joe GO, Comis LE, Berger A, Pusic I, Peer CJ, Figg WD, Cao L, Gale RP, Hakim FT, Pavletic SZ
Blood 2021 137(7): 896-907
Increased body mass index is a risk factor for acute promyelocytic leukemia
Kashanian SM, Li AY, Mustafa Ali M, Sutherland ME, Duong VH, Hambley BC, Zacholski K, El Chaer F, Holtzman NG, Imran M, Patzke CL, Cornu J, Duffy A, Dezern AE, Gojo I, Norsworthy KJ, Levis MJ, Smith BD, Baer MR, Ghiaur G, Emadi A
EJHaem 2021 2(1): 33-39
How immunodeficiency can lead to malignancy
Pai SY, Lurain K, Yarchoan R
Hematology Am Soc Hematol Educ Program 2021 2021(1): 287-295
Association of unbalanced translocation der(1;7) with germline GATA2 mutations
Kozyra EJ, Gohring G, Hickstein DD, Calvo KR, DiNardo CD, Dworzak M, de Haas V, Stary J, Hasle H, Shimamura A, Fleming MD, Inaba H, Lewis S, Hsu AP, Holland SM, Arnold DE, Mecucci C, Keel SB, Bertuch AA, Tawana K, Barzilai S, Hirabayashi S, Onozawa M, Lei S, Alaiz H, Andrikovics H, Betts D, Beverloo BH, Buechner J, Cermak M, Cervera J, Haus O, Jahnukainen K, Manola KN, Nebral K, Pasquali F, Tchinda J, Turkiewicz D, Van Roy N, Zemanova Z, Pastor VB, Strahm B, Noellke P, Niemeyer CM, Schlegelberger B, Yoshimi A, Wlodarski MW
Blood 2021 138(23): 2441-2445
Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study
Cutler C, Lee SJ, Arai S, Rotta M, Zoghi B, Lazaryan A, Ramakrishnan A, DeFilipp Z, Salhotra A, Chai-Ho W, Mehta R, Wang T, Arora M, Pusic I, Saad A, Shah NN, Abhyankar S, Bachier C, Galvin J, Im A, Langston A, Liesveld J, Juckett M, Logan A, Schachter L, Alavi A, Howard D, Waksal HW, Ryan J, Eiznhamer D, Aggarwal SK, Ieyoub J, Schueller O, Green L, Yang Z, Krenz H, Jagasia M, Blazar BR, Pavletic S
Blood 2021 138(22): 2278-2289
Clinical characterization and cytokine profile of fatigue in hematologic malignancy patients with chronic graft-versus-host disease
Goklemez S, Saligan LN, Pirsl F, Holtzman NG, Ostojic A, Steinberg SM, Hakim FT, Rose JJ, Kang Z, Yu Y, Cao L, Mitchell SA, Im A, Pavletic SZ
Bone Marrow Transplant 2021 56(12): 2934-2939
National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IIb. The 2020 Preemptive Therapy Working Group Report
Pidala J, Kitko C, Lee SJ, Carpenter P, Cuvelier GDE, Holtan S, Flowers ME, Cutler C, Jagasia M, Gooley T, Palmer J, Randolph T, Levine JE, Ayuk F, Dignan F, Schoemans H, Tkaczyk E, Farhadfar N, Lawitschka A, Schultz KR, Martin PJ, Sarantopoulos S, Inamoto Y, Socie G, Wolff D, Blazar B, Greinix H, Paczesny S, Pavletic S, Hill G
Transplant Cell Ther 2021 27(8): 632-641
Ocular surface indicators and biomarkers in chronic ocular graft-versus-host disease: a prospective cohort study
Pietraszkiewicz AA, Payne D, Abraham M, Garced A, Devarasetty KC, Wall M, Menezes SM, Ugarte S, Pirsl F, Goklemez S, Ferris FL, Barrett J, Battiwalla M, Childs RW, Pavletic SZ, Bishop RJ
Bone Marrow Transplant 2021 56(8): 1850-1858
B regulatory cells and monocyte subpopulations in patients with chronic graft-vs-host disease
Babic A, Kuric L, Zelic Kerep A, Desnica L, Lelas A, Milosevic M, Serventi-Seiwerth R, Durakovic N, Peric Z, Mravak Stipetic M, Bilic E, Ceovic R, Baresic M, Vukic T, Ljubas Kelecic D, Mazic S, Bojanic I, Hecimovic A, Bilic E, Zadro R, Vrhovac R, Pavletic SZ, Batinic D, Pulanic D
Croat Med J 2021 62(2): 154-164
Daratumumab for delayed RBC engraftment following major ABO mismatched haploidentical bone marrow transplantation
Yates B, Molloy E, Dulau-Florea A, Braylan R, Hogan L, Hickstein DD, Freeman AF, Kalsi SS, Shah NN
Transfusion 2021 61(4): 1041-1046
Case Report: Fatal Complications of BK Virus-Hemorrhagic Cystitis and Severe Cytokine Release Syndrome Following BK Virus-Specific T-Cells
Holland EM, Gonzalez C, Levy E, Valera VA, Chalfin H, Klicka-Skeels J, Yates B, Kleiner DE, Hadigan C, Dave H, Shalabi H, Hickstein DD, Su HC, Grimley M, Freeman AF, Shah NN
Front Immunol 2021 12(801281
Significant Associations of IgG Glycan Structures With Chronic Graft-Versus-Host Disease Manifestations: Results of the Cross-Sectional NIH Cohort Study
Prenc E, Pulanic D, Pucic-Bakovic M, Ugrina I, Desnica L, Milosevic M, Pirsl F, Mitchell S, Rose J, Vrhovac R, Nemet D, Lauc G, Pavletic SZ
Front Immunol 2021 12(633214
Aspartate beta-Hydroxylase (ASPH) Expression in Acute Myeloid Leukemia: A Potential Novel Therapeutic Target
Holtzman NG, Lebowitz MS, Koka R, Baer MR, Malhotra K, Shahlaee A, Ghanbari HA, Bentzen SM, Emadi A
Front Oncol 2021 11(783744
Von Willebrand Factor, Factor VIII, and Other Acute Phase Reactants as Biomarkers of Inflammation and Endothelial Dysfunction in Chronic Graft-Versus-Host Disease
Lelas A, Greinix HT, Wolff D, Eissner G, Pavletic SZ, Pulanic D
Front Immunol 2021 12(676756
The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
Arnold DE, Chellapandian D, Leiding JW
Front Immunol 2021 12(692219
Cryptosporidium infection in dedicator of cytokinesis 8 (DOCK 8) deficiency
Ben Yakov G, Sharma D, Cho MH, Shah NN, Hickstein D, Urban A, Darnell D, Kapuria D, Marko J, Kleiner DE, Hadigan CM, Danielson J, Ham H, Vittal A, Su HC, Freeman AF, Heller T
J Allergy Clin Immunol Pract 2020 8(10): 3663-3666 e3661
Sarcopenia among patients after allogeneic hematopoietic stem cell transplantation and the impact of chronic graft-versus-host disease
Ljubas Kelecic D, Lelas A, Karas I, Desnica L, Vukic T, Sabol I, Vranesic Bender D, Serventi Seiwerth R, Peric Z, Durakovic N, Vitali Cepo D, Vrhovac R, Nemet D, Pavletic S, Pulanic D, Krznaric Z
J Cancer Res Clin Oncol 2020 146(11): 2967-2978
Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases
Kahn JM, Brazauskas R, Tecca HR, Bo-Subait S, Buchbinder D, Battiwala M, Flowers MED, Savani BN, Phelan R, Broglie L, Abraham AA, Keating AK, Daly A, Wirk B, George B, Alter BP, Ustun C, Freytes CO, Beitinjaneh AM, Duncan C, Copelan E, Hildebrandt GC, Murthy HS, Lazarus HM, Auletta JJ, Myers KC, Williams KM, Page KM, Vrooman LM, Norkin M, Byrne M, Diaz MA, Kamani N, Bhatt NS, Rezvani A, Farhadfar N, Mehta PA, Hematti P, Shaw PJ, Kamble RT, Schears R, Olsson RF, Hayashi RJ, Gale RP, Mayo SJ, Chhabra S, Rotz SJ, Badawy SM, Ganguly S, Pavletic S, Nishihori T, Prestidge T, Agrawal V, Hogan WJ, Inamoto Y, Shaw BE, Satwani P
Blood Adv 2020 4(9): 2084-2094
Metabolic reprogramming augments potency of human pSTAT3-inhibited iTregs to suppress alloreactivity
Walton K, Fernandez MR, Sagatys EM, Reff J, Kim J, Lee MC, Kiluk JV, Hui JYC, McKenna D, Jr., Hupp M, Forster C, Linden MA, Lawrence NJ, Lawrence HR, Pidala J, Pavletic SZ, Blazar BR, Sebti SM, Cleveland JL, Anasetti C, Betts BC
JCI Insight 2020 5(9): e136437
Quality of Life in Patients With Skin of Color and Chronic Graft-vs-Host Disease
Smith ZI, Rosenstein RK, Banerjee S, Pichard DC, Pavletic SZ, Cowen EW
JAMA Dermatol 2020 156(5): 589-590
Immune Response Following Quadrivalent Human Papillomavirus Vaccination in Women After Hematopoietic Allogeneic Stem Cell Transplant: A Nonrandomized Clinical Trial
Stratton P, Battiwalla M, Tian X, Abdelazim S, Baird K, Barrett AJ, Cantilena CR, Childs RW, DeJesus J, Fitzhugh C, Fowler D, Gea-Banacloche J, Gress RE, Hickstein D, Hsieh M, Ito S, Kemp TJ, Khachikyan I, Merideth MA, Pavletic SZ, Quint W, Schiffman M, Scrivani C, Shanis D, Shenoy AG, Struijk L, Tisdale JF, Wagner S, Williams KM, Yu Q, Wood LV, Pinto LA
JAMA Oncol 2020 6(5): 696-705
NCCN Guidelines: Pretransplant Recipient Evaluation and Management of Graft-Versus-Host Disease
Cutler C, Pavletic SZ
J Natl Compr Canc Netw 2020 18(5): 645-647
Sequencing of RNA in single cells reveals a distinct transcriptome signature of hematopoiesis in GATA2 deficiency
Wu Z, Gao S, Diamond C, Kajigaya S, Chen J, Shi R, Palmer C, Hsu AP, Calvo KR, Hickstein DD, Holland SM, Young NS
Blood Adv 2020 4(12): 2656-2670
Calcinosis Cutis in the Setting of Chronic Skin Graft-Versus-Host Disease
Saardi KM, Rosenstein RK, Anadkat MJ, Micheletti RG, Schiffenbauer AI, Pavletic SZ, Cowen EW
JAMA Dermatol 2020 156(7): 814-817
Refined National Institutes of Health response algorithm for chronic graft-versus-host disease in joints and fascia
Inamoto Y, Lee SJ, Onstad LE, Flowers MED, Hamilton BK, Jagasia MH, Martin PJ, Pavletic SZ, Pidala JA, Pusic I, Vogelsang GB, Wolff D, Carpenter PA
Blood Adv 2020 4(1): 40-46
Safety and efficacy of CAR T cells in a patient with lymphoma and a coexisting autoimmune neuropathy
Jhaveri KS, Schlam I, Holtzman NG, Peravali M, Richardson PK, Dahiya S, Malkovska V, Rapoport AP
Blood Adv 2020 4(23): 6019-6022
A New Standard in Graft-versus-Host Disease Prophylaxis? An Introduction to Blood and Marrow Transplant Clinical Trials Network 1703
DeFilipp Z, Burns LJ, Jaglowski SM, Leppin AL, Pavletic S, Waldman B, Weisdorf DJ, Wood WA, Khera N
Biol Blood Marrow Transplant 2020 26(12): e305-e308
Risk Factors for Graft-versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation Using Post-Transplant Cyclophosphamide
Im A, Rashidi A, Wang T, Hemmer M, MacMillan ML, Pidala J, Jagasia M, Pavletic S, Majhail NS, Weisdorf D, Abdel-Azim H, Agrawal V, Al-Homsi AS, Aljurf M, Askar M, Auletta JJ, Bashey A, Beitinjaneh A, Bhatt VR, Byrne M, Cahn JY, Cairo M, Castillo P, Cerny J, Chhabra S, Choe H, Ciurea S, Daly A, Perez MAD, Farhadfar N, Gadalla SM, Gale R, Ganguly S, Gergis U, Hanna R, Hematti P, Herzig R, Hildebrandt GC, Lad DP, Lee C, Lehmann L, Lekakis L, Kamble RT, Kharfan-Dabaja MA, Khandelwal P, Martino R, Murthy HS, Nishihori T, O'Brien TA, Olsson RF, Patel SS, Perales MA, Prestidge T, Qayed M, Romee R, Schoemans H, Seo S, Sharma A, Solh M, Strair R, Teshima T, Urbano-Ispizua A, Van der Poel M, Vij R, Wagner JL, William B, Wirk B, Yared JA, Spellman SR, Arora M, Hamilton BK
Biol Blood Marrow Transplant 2020 26(8): 1459-1468
Motor ability, function, and health-related quality of life as correlates of symptom burden in patients with sclerotic chronic graft-versus-host disease receiving imatinib mesylate
Rosenthal EA, Ho PS, Joe GO, Mitchell SA, Booher S, Pavletic SZ, Baird K, Cowen EW, Comis LE
Support Care Cancer 2020 28(8): 3679-3689
Clinical characteristics and cytokine biomarkers in patients with chronic graft-vs-host disease persisting seven or more years after diagnosis
Goklemez S, Im AP, Cao L, Pirsl F, Steinberg SM, Curtis LM, Mitchell SA, Cowen EW, Baruffaldi J, Rose J, Mays J, Ostojic A, Holtzman NG, Hakim FT, Pavletic SZ
Am J Hematol 2020 95(4): 387-394
Toxoplasma-induced hemophagocytic lymphohistiocytosis after haploidentical allogeneic stem cell transplantation
Sanchez-Petitto G, Holtzman NG, Bukhari A, Brown M, Morales MK, Koka M, Yared JA, Dahiya S, Rapoport AP, Hardy NM
Transpl Infect Dis 2020 22(2): e13242
Autoimmunity Following Allogeneic Hematopoietic Stem Cell Transplantation
Buxbaum NP, Pavletic SZ
Front Immunol 2020 11(2017)
Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency
Freeman AF, Yazigi N, Shah NN, Kleiner DE, Parta M, Atkinson P, Heller T, Holland SM, Kaufman SS, Khan KM, Hickstein DD
Transplantation 2019 103(10): 2144-2149
Clinical utilization of Chimeric Antigen Receptor T-cells (CAR-T) in B-cell acute lymphoblastic leukemia (ALL)-an expert opinion from the European Society for Blood and Marrow Transplantation (EBMT) and the American Society for Blood and Marrow Transplant
Kansagra AJ, Frey NV, Bar M, Laetsch TW, Carpenter PA, Savani BN, Heslop HE, Bollard CM, Komanduri KV, Gastineau DA, Chabannon C, Perales MA, Hudecek M, Aljurf M, Andritsos L, Barrett JA, Bachanova V, Bonini C, Ghobadi A, Gill SI, Hill JA, Kenderian S, Kebriaei P, Nagler A, Maloney D, Liu HD, Shah NN, Kharfan-Dabaja MA, Shpall EJ, Mufti GJ, Johnston L, Jacoby E, Bazarbachi A, DiPersio JF, Pavletic SZ, Porter DL, Grupp SA, Sadelain M, Litzow MR, Mohty M, Hashmi SK
Bone Marrow Transplant 2019 54(11): 1868-1880
Accompanying editorial on paper Neutrophil extracellular traps (NETs) contribute to Pathological Changes of ocular graft-vs.-Host Disease (oGVHD) dry eye: Implications for novel Biomarkers and Therapeutic Strategies by Seungwon An et al
Petricek I, Pavletic SZ
Ocul Surf 2019 17(3): 372-373
Use of Chimeric Antigen Receptor T Cell Therapy in Clinical Practice for Relapsed/Refractory Aggressive B Cell Non-Hodgkin Lymphoma: An Expert Panel Opinion from the American Society for Transplantation and Cellular Therapy
Jain T, Bar M, Kansagra AJ, Chong EA, Hashmi SK, Neelapu SS, Byrne M, Jacoby E, Lazaryan A, Jacobson CA, Ansell SM, Awan FT, Burns L, Bachanova V, Bollard CM, Carpenter PA, DiPersio JF, Hamadani M, Heslop HE, Hill JA, Komanduri KV, Kovitz CA, Lazarus HM, Serrette JM, Mohty M, Miklos D, Nagler A, Pavletic SZ, Savani BN, Schuster SJ, Kharfan-Dabaja MA, Perales MA, Lin Y
Biol Blood Marrow Transplant 2019 25(12): 2305-2321
Oral Complications of Chronic Graft-Versus-Host Disease
Fall-Dickson JM, Pavletic SZ, Mays JW, Schubert MM
J Natl Cancer Inst Monogr 2019 2019(53):
GATA2 Deficiency: Early Identification for Improved Clinical Outcomes
Cole K, Avila D, Parta M, Schuver B, Holland S, Shah N, Hickstein D
Clin J Oncol Nurs 2019 23(4): 417-422