title: Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.
abstract: We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.
authors.affiliation: Clinical Research Unit, Eurocord office, Hôpital Saint Louis APHP, Paris, France. annalisa.ruggeri@eurocord.org
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
authors.forename: Andromachi
authors.initials: A
authors.lastname: Scaravadou
authors.forename: Mitchell S
authors.initials: MS
authors.lastname: Cairo
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: John R
authors.initials: JR
authors.lastname: Wingard
authors.forename: Anders
authors.initials: A
authors.lastname: Fasth
authors.forename: Luca
authors.initials: L
authors.lastname: Lo Nigro
authors.forename: Mouhab
authors.initials: M
authors.lastname: Ayas
authors.forename: Duncan
authors.initials: D
authors.lastname: Purtill
authors.forename: Karim
authors.initials: K
authors.lastname: Boudjedir
authors.forename: Wagnara
authors.initials: W
authors.lastname: Chaves
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.forename: John
authors.initials: J
authors.lastname: Wagner
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D000755:Anemia, Sickle Cell / Q000401:mortality / Q000628:therapy*; D002452:Cell Count; D002648:Child; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000706:statistics & numerical data; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D006650:Histocompatibility Testing; D006801:Humans; D008297:Male; D009504:Neutrophils; D012042:Registries; D016019:Survival Analysis; D013789:Thalassemia / Q000401:mortality / Q000628:therapy*; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome