Hematopoietic Cell Transplantation with Reduced Intensity Conditioning Using Fludarabine/Busulfan or Fludarabine/Melphalan for Primary Immunodeficiency Diseases.
Akira NishimuraYuki AokiYasuyoshi IshiwataTakuya IchimuraJunichi UeyamaYuta KawaharaTakahiro TomodaMaiko InoueKazuaki MatsumotoKento InoueHaruka HirokiShintaro OnoMotoi YamashitaTsubasa OkanoMari Tanaka-KubotaMiho AshiaraiSatoshi MiyamotoReiji MiyawakiChika YamagishiMari TezukaTeppei OkawaAkihiro HoshinoAkifumi EndoMasato YasuharaTakahiro KamiyaNoriko MitsuikiToshiaki OnoTakeshi IsodaMasakatsu YanagimachiDaisuke TomizawaMasayuki NagasawaShuki MizutaniMichiko KajiwaraMasatoshi TakagiHirokazu KaneganeKohsuke ImaiTomohiro MorioPublished in: Journal of clinical immunology (2021)
RIC-HCT with FluBU is a safe and effective strategy for obtaining high-level donor chimerism, immune reconstitution including B cell function, and long-term survival in patients with SCID. In patients with non-SCID PID, the results varied according to the subtype of the disease. Further prospective studies are required to optimize the conditioning regimen for non-SCID PID.