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 × 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.
| Original language | English |
|---|---|
| Pages (from-to) | 2137-2143 |
| Number of pages | 7 |
| Journal | Blood |
| Volume | 101 |
| Issue number | 6 |
| DOIs | |
| Publication status | Published - Mar 15 2003 |
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ASJC Scopus subject areas
- Hematology
Cite this
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. / Locatelli, Franco; Rocha, Vanderson; Reed, William; Bernaudin, Françoise; Ertem, Mehmet; Grafakos, Stelios; Brichard, Benedicte; Li, Xiaxin; Nagler, Arnon; Giorgiani, Giovanna; Haut, Paul R.; Brochstein, Joel A.; Nugent, Diane J.; Blatt, Julie; Woodard, Paul; Kurtzberg, Joanne; Rubin, Charles M.; Miniero, Roberto; Lutz, Patrick; Raja, Thirumalairaj; Roberts, Irene; Will, Andrew M.; Yaniv, Isaac; Vermylen, Christiane; Tannoia, Nunzia; Garnier, Federico; Ionescu, Irina; Walters, Mark C.; Lubin, Bertram H.; Gluckman, Eliane.
In: Blood, Vol. 101, No. 6, 15.03.2003, p. 2137-2143.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease
AU - Locatelli, Franco
AU - Rocha, Vanderson
AU - Reed, William
AU - Bernaudin, Françoise
AU - Ertem, Mehmet
AU - Grafakos, Stelios
AU - Brichard, Benedicte
AU - Li, Xiaxin
AU - Nagler, Arnon
AU - Giorgiani, Giovanna
AU - Haut, Paul R.
AU - Brochstein, Joel A.
AU - Nugent, Diane J.
AU - Blatt, Julie
AU - Woodard, Paul
AU - Kurtzberg, Joanne
AU - Rubin, Charles M.
AU - Miniero, Roberto
AU - Lutz, Patrick
AU - Raja, Thirumalairaj
AU - Roberts, Irene
AU - Will, Andrew M.
AU - Yaniv, Isaac
AU - Vermylen, Christiane
AU - Tannoia, Nunzia
AU - Garnier, Federico
AU - Ionescu, Irina
AU - Walters, Mark C.
AU - Lubin, Bertram H.
AU - Gluckman, Eliane
PY - 2003/3/15
Y1 - 2003/3/15
N2 - 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.
AB - 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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U2 - 10.1182/blood-2002-07-2090
DO - 10.1182/blood-2002-07-2090
M3 - Article
VL - 101
SP - 2137
EP - 2143
JO - Blood
JF - Blood
SN - 0006-4971
IS - 6
ER -