title: Allogeneic hematopoietic stem cell transplantation following reduced-intensity conditioning regimen in children: a single-center experience.
abstract: This single-center retrospective study reported the outcome of 19 children treated with a reduced-intensity conditioning (RIC) regimen prior to allogeneic stem cell transplantation (allo-SCT), for hematologic malignancies (n = 17), bone marrow failure (n = 1), and neuroblastoma (n = 1). Children were ineligible for standard myeloablative conditioning because of severe comorbidities (n = 9), a previous auto or allo-SCT (n = 7) or a prior history of extensive chemotherapy (n = 3). All patients underwent a fludarabine-based RIC regimen, and received grafts from matched-related donors (n = 5), match-unrelated donors (n = 6), or unrelated cord blood (UCB, n = 8). In this series, two patients treated with UCB failed to engraft and 63% achieved full donor chimerism at day 100 after allo-SCT. With a median follow-up of 537 d (range, 115-4136), treatment-related mortality was 16% and overall survival was 47%. The principal cause of death was disease relapse (n = 7). Acute graft versus host disease (GVHD) occurred in 53% of patients, while only 10% developed extensive chronic GVHD. Overall, results from this series suggest that RIC allo-SCT can be a valid alternative treatment option in unfit children with malignant hematological diseases. Prospective studies are needed to enlarge pediatric experience in this domain and better identify those children more suitable for a RIC allo-SCT approach.
authors.affiliation: Centre Hospitalier et Universitaire (CHU) de Nantes, Service d'Onco-Hématologie Pédiatrique, Nantes, France.
authors.forename: Marion
authors.initials: M
authors.lastname: Strullu
authors.forename: Fanny
authors.initials: F
authors.lastname: Rialland
authors.forename: Xavier
authors.initials: X
authors.lastname: Cahu
authors.forename: Eolia
authors.initials: E
authors.lastname: Brissot
authors.forename: Nadege
authors.initials: N
authors.lastname: Corradini
authors.forename: Caroline
authors.initials: C
authors.lastname: Thomas
authors.forename: Nicolas
authors.initials: N
authors.lastname: Blin
authors.forename: Xavier
authors.initials: X
authors.lastname: Rialland
authors.forename: Françoise
authors.initials: F
authors.lastname: Méchinaud
authors.forename: Mohamad
authors.initials: M
authors.lastname: Mohty
mesh_terms: D000293:Adolescent; D001855:Bone Marrow Diseases / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D019337:Hematologic Neoplasms / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D009447:Neuroblastoma / Q000628:therapy; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D061349:Unrelated Donors