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Page 1
Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity.
Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder DK, Cattoni A, Chernyshova L, Ciznar P, Cole T, Czogala W, Dueckers G, Edgar JDM, Erbey F, Fasth A, Ferrua F, Formankova R, Gambineri E, Gennery AR, Goldman FD, Gonzalez-Granado LI, Heilmann C, Heiskanen-Kosma T, Juntti H, Kainulainen L, Kanegane H, Karaca NE, Kilic SS, Klein C, Koltan S MD, PhD, Kondratenko I, Meyts I, Nasrullayeva GM, Notarangelo LD, Pasic S, Pellier I, Pignata C, Misbah SA, Schulz AS, Segundo GR, Shcherbina A, Slatter MA, Sokolic R, Soler-Palacin P, Stepensky P, van Montfrans JM, Ryhänen S, Wolska-Kuśnierz B MD, PhD, Ziegler JB, Zhao X, Aiuti A, Ochs HD, Albert MH. Vallée TC, et al. Among authors: stepensky p. Blood. 2024 Apr 5:blood.2023021411. doi: 10.1182/blood.2023021411. Online ahead of print. Blood. 2024. PMID: 38579284
Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study.
Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, Vermont C, Ahmad I, Shaw PJ, Telles da Cunha JM, Schlegel PG, Hough R, Fasth A, Kentouche K, Gruhn B, Fernandes JF, Lachance S, Bredius R, Resnick IB, Belohradsky BH, Gennery A, Fischer A, Gaspar HB, Schanz U, Seger R, Rentsch K, Veys P, Haddad E, Albert MH, Hassan M; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation. Güngör T, et al. Among authors: stepensky p. Lancet. 2014 Feb 1;383(9915):436-48. doi: 10.1016/S0140-6736(13)62069-3. Epub 2013 Oct 23. Lancet. 2014. PMID: 24161820 Clinical Trial.
Transplant results in adults with Fanconi anaemia.
Bierings M, Bonfim C, Peffault De Latour R, Aljurf M, Mehta PA, Knol C, Boulad F, Tbakhi A, Esquirol A, McQuaker G, Sucak GA, Othman TB, Halkes CJM, Carpenter B, Niederwieser D, Zecca M, Kröger N, Michallet M, Risitano AM, Ehninger G, Porcher R, Dufour C; EBMT SAA WP. Bierings M, et al. Br J Haematol. 2018 Jan;180(1):100-109. doi: 10.1111/bjh.15006. Epub 2017 Nov 2. Br J Haematol. 2018. PMID: 29094350 Free article.
Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma.
Kfir-Erenfeld S, Asherie N, Lebel E, Vainstein V, Assayag M, Dubnikov Sharon T, Grisariu S, Avni B, Elias S, Alexander-Shani R, Bessig N, Shehadeh A, Ishtay A, Zelmanovich V, Zimran E, Pick M, Roziner I, Kenett RS, Cohen YC, Avivi I, Cohen CJ, Gatt MEE, Stepensky P. Kfir-Erenfeld S, et al. Among authors: stepensky p. Blood Adv. 2024 May 20:bloodadvances.2024012967. doi: 10.1182/bloodadvances.2024012967. Online ahead of print. Blood Adv. 2024. PMID: 38768428
Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP.
Wachowiak J, Galimard JE, Dalissier A, Rihani R, AlSaedi H, Wynn RF, Dalle JH, Peffault de Latour R, Sedlacek P, Balduzzi A, Schroeder T, Bodova I, Gonzalez Vicent M, Gruhn B, Hamladji RM, Krivan G, Patrick K, Sobkowiak-Sobierajska A, Stepensky P, Unal A, Amrolia P, Perez Martinez A, Rialland F, Aljurf M, Isgro A, Toren A, Bierings M, Corbacioglu S, Kałwak K. Wachowiak J, et al. Among authors: stepensky p. Bone Marrow Transplant. 2024 Apr 16. doi: 10.1038/s41409-024-02286-3. Online ahead of print. Bone Marrow Transplant. 2024. PMID: 38627449
Development and Validation of a Prediction Model of Outcome After B-Cell Maturation Antigen-Directed Chimeric Antigen Receptor T-Cell Therapy in Relapsed/Refractory Multiple Myeloma.
Gagelmann N, Dima D, Merz M, Hashmi H, Ahmed N, Tovar N, Oliver-Caldés A, Stölzel F, Rathje K, Fischer L, Born P, Schäfer L, Albici AM, Schub N, Kfir-Erenfeld S, Assayag M, Asherie N, Wulf GG, Kharboutli S, Müller F, Shune L, Davis JA, Anwer F, Vucinic V, Platzbecker U, Ayuk F, Kröger N, Khouri J, Gurnari C, McGuirk J, Stepensky P, Abdallah AO, Fernández de Larrea C. Gagelmann N, et al. Among authors: stepensky p. J Clin Oncol. 2024 May 10;42(14):1665-1675. doi: 10.1200/JCO.23.02232. Epub 2024 Feb 15. J Clin Oncol. 2024. PMID: 38358946 Free PMC article.
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Migliavacca M, Barzaghi F, Fossati C, Rancoita PMV, Gabaldo M, Dionisio F, Giannelli S, Salerio FA, Ferrua F, Tucci F, Calbi V, Gallo V, Recupero S, Consiglieri G, Pajno R, Sambuco M, Priolo A, Ferri C, Garella V, Monti I, Silvani P, Darin S, Casiraghi M, Corti A, Zancan S, Levi M, Cesana D, Carlucci F, Pituch-Noworolska A, AbdElaziz D, Baumann U, Finocchi A, Cancrini C, Ladogana S, Meinhardt A, Meyts I, Montin D, Notarangelo LD, Porta F, Pasquet M, Speckmann C, Stepensky P, Tommasini A, Rabusin M, Karakas Z, Galicchio M, Leonardi L, Duse M, Guner SN, Di Serio C, Ciceri F, Bernardo ME, Aiuti A, Cicalese MP. Migliavacca M, et al. Among authors: stepensky p. Nat Med. 2024 Feb;30(2):488-497. doi: 10.1038/s41591-023-02789-4. Epub 2024 Feb 14. Nat Med. 2024. PMID: 38355973
Management of Atopy with Dupilumab and Omalizumab in CADINS Disease.
Diaz-Cabrera NM, Bauman BM, Iro MA, Dabbah-Krancher G, Molho-Pessach V, Zlotogorski A, Shamriz O, Dinur-Schejter Y, Sharon TD, Stepensky P, Tal Y, Eisenstein EM, Pietzsch L, Schuetz C, Abreu D, Coughlin CC, Cooper MA, Milner JD, Williams A, Armoni-Weiss G, Snow AL, Leiding JW. Diaz-Cabrera NM, et al. Among authors: stepensky p. J Clin Immunol. 2024 Jan 17;44(2):48. doi: 10.1007/s10875-023-01636-y. J Clin Immunol. 2024. PMID: 38231347
Combined Immunodeficiency Caused by a Novel Nonsense Mutation in LCK.
Keller B, Kfir-Erenfeld S, Matusewicz P, Hartl F, Lev A, Lee YN, Simon AJ, Stauber T, Elpeleg O, Somech R, Stepensky P, Minguet S, Schraven B, Warnatz K. Keller B, et al. Among authors: stepensky p. J Clin Immunol. 2023 Dec 19;44(1):4. doi: 10.1007/s10875-023-01614-4. J Clin Immunol. 2023. PMID: 38112969 Free PMC article.
Chronic graft-versus-host disease detected by tissue-specific cell-free DNA methylation biomarkers.
Avni B, Neiman D, Shaked E, Gal-Rosenberg O, Grisariu S, Kuzli M, Avni I, Fracchia A, Stepensky P, Zuckerman T, Lev-Sagie A, Fox-Fisher I, Piyanzin S, Moss J, Salpeter SJ, Glaser B, Shemer R, Dor Y. Avni B, et al. Among authors: stepensky p. J Clin Invest. 2024 Jan 16;134(2):e163541. doi: 10.1172/JCI163541. J Clin Invest. 2024. PMID: 37971879 Free PMC article.
174 results