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Prepublished online as a Blood First Edition Paper on November 7, 2002; DOI 10.1182/blood-2002-07-2090.
Submitted July 12, 2002
Accepted October 25, 2002
Related umbilical cord blood transplant in patients with Thalassemia and Sickle Cell Disease
Franco Locatelli*, Vanderson Rocha, William Reed, Francoise 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
IRCCS Policlinico San Matteo, Oncoematologia Pediatrica, Pavia, Italy
Hopital Saint-Louis, BMT Unit and Eurocord Office, Paris, France
Sibling Donor Cord Blood Program, Children's Hospital Oakland Research Institute, Oakland, CA, USA
Ibni Sina Hospital, Ankara, Turkey
Aghia Sophia Children's Hospital, Athens, Greece
Cliniques St. Luc, Brussels, Belgium
Hospital Nanfang, Guangzhou, China
The Chaim Sheba Medical Center, Tel Hashomer, Israel
Indiana University School of Medicine, Indianapolis, IN, USA
Hackensack University Medical Center, Hackensack, NJ, USA
Children's Hospital of Orange County, Orange, NJ, USA
University of North Carolina, Chapel Hill, NC, USA
St. Jude Children's Hospital, Memphis, TN, USA
Duke University Medical Center, Durham, NC, USA
Department of Pediatrics, University of Chicago, Chicago, IL, USA
Ospedale San Luigi di Orbassano, Turin, Italy
Hopital de Hautpierre, CHU, Strasbourg, France
Apollo Speciality Hospital, Chennai, India
Hammersmith Hospital, London, United Kingdom
Royal Manchester Children's Hospital, Pendlebury, United Kingdom
Schneider Children's Med. Center of Israel, Petach-Tikva, Israel
Deartment of Hematology II, University of Bari, Bari, Italy
Division of Blood and Marrow Transplantation, Children's Hospital Oakland, Oakland, CA, USA
* Corresponding author; email: f.locatelli{at}smatteo.pv.it.
Background: 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 a not negligible risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (cGVHD). Patients and methods: We analyzed 44 patients (median age 5 years, range 1-20) given an allogeneic related cord blood transplantation (CBT) for either thalassemia (n=33) or SCD (n=11). Thirty children were given Cyclosporine A (Cs-A) alone as GVHD prophylaxis, 10 received Cs-A and Methotrexate (MTX) and 4 patients received other combinations of immunosuppressive drugs. Median number of nucleated cells infused was 4.0 x 107/kg (range 1.2-10). Results: No patient died and 36 out of 44 children remain disease-free, with a median follow-up of 24 months (range 4-76). Only one patient with SCD did not have sustained donor engraftment as compared to 7 out 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 II acute GVHD; only 2 out of the 36 patients at risk developed limited cGVHD. 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.
Conclusions: Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplant strategies could further improve these results.
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