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Prepublished online as a Blood First Edition Paper on November 7, 2002; DOI 10.1182/blood-2002-07-2090.
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Blood, 15 March 2003, Vol. 101, No. 6, pp. 2137-2143
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Related umbilical cord blood transplantation in patients with
thalassemia and sickle cell disease
Franco Locatelli,
Vanderson Rocha,
William Reed,
Françoise Bernaudin,
Mehmet Ertem,
Stelios Grafakos,
Benedicte Brichard,
Xiaxin Li,
Arnon Nagler,
Giovanna Giorgiani,
Paul R. Haut,
Joel A. Brochstein,
Diane J. Nugent,
Julie Blatt,
Paul Woodard,
Joanne Kurtzberg,
Charles M. Rubin,
Roberto Miniero,
Patrick Lutz,
Thirumalairaj Raja,
Irene Roberts,
Andrew M. Will,
Isaac Yaniv,
Christiane Vermylen,
Nunzia Tannoia,
Federico Garnier,
Irina Ionescu,
Mark C. Walters,
Bertram
H. Lubin, and
Eliane Gluckman for
the Eurocord Transplant Group
From the Oncoematologia Pediatrica, Istituto di
Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo,
Università di Pavia, Italy; Hôpital Saint
Louis and Eurocord office, Paris, France; Sibling Donor
Cord Blood Program, Children's Hospital Oakland Research Institute,
CA; Ibni Sina Hospital, Ankara, Turkey; Aghia Sophia
Children's Hospital, Athens, Greece; Cliniques St. Luc,
Brussels, Belgium; Hospital Nanfang, Guangzhou, People's
Republic of China; The Chaim Sheba Medical Center, Tel Hashomer,
Israel; Indiana University School of Medicine,
Indianapolis; Hackensack University Medical Center, NJ; Children's
Hospital of Orange County, Orange, CA; University of North Carolina,
Chapel Hill; St Jude Children's Research Hospital, Memphis, TN; Duke
University Medical Center, Durham, NC; Department of Pediatrics,
University of Chicago, Chicago, IL; University of Turin, Ospedale San
Luigi di Orbassano, Torino, Italy; Hôpital de
Hautepierre, Centre Hospitalier Universitaire, Strasbourg,
France; Apollo Specialty Hospital, Chennai,
India; Hammersmith Hospital, Royal Postgraduate Medical
School, London, United Kingdom; Royal Manchester
Children's Hospital, Pendlebury, United Kingdom;
Schneider Children's Medical Center of Israel, Petach-Tikva;
Department of Hematology II, University of Bari, Italy;
and Division of Blood and Marrow Transplantation, Children's Hospital,
Oakland, CA.
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 × 107/kg (range, 1.2-10 ×
107/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.
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