Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.
СтатьяНазвание и аннотация: Перевод в очереди
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Alexander Jessie L, Dávila Saldaña Blachy J, Brazauskas Ruta, Dammalapati Sravya Gethika, Griffith Linda M, Shah Ami J, Shimano Kristin A, Ochs Hans D, Bleesing Jack J, Ebens Christen L, Kapadia Malika, Bauchat Andrea, Kapoor Neena, Oved Joseph H, Eissa Hesham, Lust Hannah, Keller Michael D, Haines Hilary, Chandrakasan Shanmuganathan, Talano Julie-An, Rayes Ahmad, Madden Lisa, Shereck Evan, Miller Holly K, Satter Lisa Forbes, Martinez Caridad, Rozmus Jacob, Bednarski Jeffrey J, Yu Lolie C, Chellapandian Deepakbabu, Aquino Victor M, Knutsen Alan, Chong Hey, Chopek Ashley, Gillio Alfred P, Joshi Avni, Rangarajan Hemalatha, Moore Theodore B, Andolina Jeffrey R, DeSantes Kenneth B, Vander Lugt Mark, Prockop Susan E, Shyr David C, Sullivan Kathleen E, Parikh Suhag, Weinacht Katja G, Torgerson Troy R, Marsh Rebecca, Dvorak Christopher C, Chan Alice Y, Haddad Elie, Heimall Jennifer R, Pulsipher Michael A, Leiding Jennifer W, Kohn Donald B, Puck Jennifer M, Notarangelo Luigi D, Rawlings David J, Cowan Morton J, Petrovic Aleksandra, Pai Sung-Yun, Burroughs Lauri M
Аннотация
Wiskott-Aldrich syndrome (WAS), an X-linked disorder characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancy, can be effectively treated with allogeneic hematopoietic cell transplantation (HCT). Older age at HCT and mismatched donors are known to affect overall survival (OS). However, the influence of specific clinical manifestations or WAS variant class on OS and factors associated with event-free survival (EFS) remain incompletely defined. We analyzed outcomes of 308 patients with WAS who underwent HCT at 37 institutions of the Primary Immune Deficiency Treatment Consortium from 1990 to 2018. With a median follow-up of 5.3 years, the 5-year OS and EFS were 87.2% and 79.7%, respectively. Age ≥5 years, donor type, and a pre-HCT history of severe infection had a negative impact on OS and EFS, whereas pre-HCT autoimmunity had no impact. Reduced-intensity regimens were associated with lower T-cell and myeloid donor chimerism, particularly when non-busulfan-based regimens were used. Low myeloid donor chimerism was associated with lower platelet counts. Mixed chimerism was not consistently associated with post-HCT autoimmunity. Patients with class I (exon 1-2 missense and intron 5 hot spot variants) and class II variants (all others) had similar pre-HCT clinical symptom severity and no difference in OS, EFS, or platelet recovery post-HCT. In conclusion, our study showed excellent long-term OS and EFS after HCT for WAS, highlighting the importance of early HCT, before the development of severe infections. We confirmed that HCT using busulfan-based conditioning was associated with improved donor chimerism and platelet recovery. This trial was registered at www.clinicaltrials.gov as NCT02064933.