Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
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.
Locatelli
Franco
F
Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Università di Pavia, Italy. f.locatelli@smatteo.pv.it
Rocha
Vanderson
V
Reed
William
W
Bernaudin
Françoise
F
Ertem
Mehmet
M
Grafakos
Stelios
S
Brichard
Benedicte
B
Li
Xiaxin
X
Nagler
Arnon
A
Giorgiani
Giovanna
G
Haut
Paul R
PR
Brochstein
Joel A
JA
Nugent
Diane J
DJ
Blatt
Julie
J
Woodard
Paul
P
Kurtzberg
Joanne
J
Rubin
Charles M
CM
Miniero
Roberto
R
Lutz
Patrick
P
Raja
Thirumalairaj
T
Roberts
Irene
I
Will
Andrew M
AM
Yaniv
Isaac
I
Vermylen
Christiane
C
Tannoia
Nunzia
N
Garnier
Federico
F
Ionescu
Irina
I
Walters
Mark C
MC
Lubin
Bertram H
BH
Gluckman
Eliane
E
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
D007166:Immunosuppressive Agents; D016572:Cyclosporine; D008727:Methotrexate