title: Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
abstract: Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 x 10(7)/kg (range, 1.2-10 x 10(7)/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79% and 90% for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.
authors.affiliation: Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Università di Pavia, Italy. f.locatelli@smatteo.pv.it
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
authors.forename: William
authors.initials: W
authors.lastname: Reed
authors.forename: Françoise
authors.initials: F
authors.lastname: Bernaudin
authors.forename: Mehmet
authors.initials: M
authors.lastname: Ertem
authors.forename: Stelios
authors.initials: S
authors.lastname: Grafakos
authors.forename: Benedicte
authors.initials: B
authors.lastname: Brichard
authors.forename: Xiaxin
authors.initials: X
authors.lastname: Li
authors.forename: Arnon
authors.initials: A
authors.lastname: Nagler
authors.forename: Giovanna
authors.initials: G
authors.lastname: Giorgiani
authors.forename: Paul R
authors.initials: PR
authors.lastname: Haut
authors.forename: Joel A
authors.initials: JA
authors.lastname: Brochstein
authors.forename: Diane J
authors.initials: DJ
authors.lastname: Nugent
authors.forename: Julie
authors.initials: J
authors.lastname: Blatt
authors.forename: Paul
authors.initials: P
authors.lastname: Woodard
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: Charles M
authors.initials: CM
authors.lastname: Rubin
authors.forename: Roberto
authors.initials: R
authors.lastname: Miniero
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Thirumalairaj
authors.initials: T
authors.lastname: Raja
authors.forename: Irene
authors.initials: I
authors.lastname: Roberts
authors.forename: Andrew M
authors.initials: AM
authors.lastname: Will
authors.forename: Isaac
authors.initials: I
authors.lastname: Yaniv
authors.forename: Christiane
authors.initials: C
authors.lastname: Vermylen
authors.forename: Nunzia
authors.initials: N
authors.lastname: Tannoia
authors.forename: Federico
authors.initials: F
authors.lastname: Garnier
authors.forename: Irina
authors.initials: I
authors.lastname: Ionescu
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.forename: Bertram H
authors.initials: BH
authors.lastname: Lubin
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
mesh_terms: D000208:Acute Disease; D000755:Anemia, Sickle Cell / Q000628:therapy*; D001792:Blood Platelets; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D036101:Cord Blood Stem Cell Transplantation; D016572:Cyclosporine / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D009504:Neutrophils; D015996:Survival Rate; D013789:Thalassemia / Q000628:therapy*; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D016572:Cyclosporine; D008727:Methotrexate