Abstract
Outcomes remain poor for refractory severe aplastic anemia (SAA) patients. Alternative donor transplantation may be considered, but results from previous studies are not encouraging. We conducted a prospective nationwide phase 2 study to assess unrelated cord blood (CB) transplantation (CBT) efficacy and safety in refractory SAA patients (Aplastic Anemia and Cord Blood Transplantation protocol). To demonstrate a significant difference in 1-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients and therefore included 26 (median age, 16 years). Eligibility criteria required 1 or 2 unrelated CB units, containing separately or together >4 × 107 frozen nucleated cells (NCs) per kilogram of recipient body weight. Conditioning regimen comprised fludarabine (FLU), cyclophosphamide (CY), antithymocyte globulin (ATG), and 2-Gy total body irradiation (TBI). With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. Twenty-three patients were alive at 1 year, with an 88.5% overall survival (OS) rate, differing significantly from the expected 20% (P < .0001; 84% OS at 2 years). CBT with units containing ≥4 × 107 frozen NCs per kilogram is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors. This trial was registered at www.clinicaltrials.gov as #NCT01343953.
| Original language | English |
|---|---|
| Pages (from-to) | 750-754 |
| Number of pages | 5 |
| Journal | Blood |
| Volume | 132 |
| Issue number | 7 |
| DOIs | |
| Publication status | Published - Aug 16 2018 |
| Externally published | Yes |
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Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia : a nationwide phase 2 study. / Francophone Society of Bone Marrow Transplantation and Cellular Therapy.
In: Blood, Vol. 132, No. 7, 16.08.2018, p. 750-754.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia
T2 - a nationwide phase 2 study
AU - Francophone Society of Bone Marrow Transplantation and Cellular Therapy
AU - Peffault de Latour, Regis
AU - Chevret, Sylvie
AU - Jubert, Charlotte
AU - Sirvent, Anne
AU - Galambrun, Claire
AU - Ruggeri, Annalisa
AU - Gandemer, Virginie
AU - Cornillon, Jérôme
AU - Rialland, Fanny
AU - Dalle, Jean-Hugues
AU - Forcade, Edouard
AU - Bruno, Benedicte
AU - Paillard, Catherine
AU - Rorlich, Pierre S
AU - Salmon, Alexandra
AU - Fürst, Sabine
AU - Sicre de Fontbrune, Flore
AU - Rubio, Marie Therese
AU - Bay, Jacques-Olivier
AU - Mohty, Mohamad
AU - Larghero, Jerome
AU - Gluckman, Eliane
AU - Socié, Gerard
N1 - © 2018 by The American Society of Hematology.
PY - 2018/8/16
Y1 - 2018/8/16
N2 - Outcomes remain poor for refractory severe aplastic anemia (SAA) patients. Alternative donor transplantation may be considered, but results from previous studies are not encouraging. We conducted a prospective nationwide phase 2 study to assess unrelated cord blood (CB) transplantation (CBT) efficacy and safety in refractory SAA patients (Aplastic Anemia and Cord Blood Transplantation protocol). To demonstrate a significant difference in 1-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients and therefore included 26 (median age, 16 years). Eligibility criteria required 1 or 2 unrelated CB units, containing separately or together >4 × 107 frozen nucleated cells (NCs) per kilogram of recipient body weight. Conditioning regimen comprised fludarabine (FLU), cyclophosphamide (CY), antithymocyte globulin (ATG), and 2-Gy total body irradiation (TBI). With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. Twenty-three patients were alive at 1 year, with an 88.5% overall survival (OS) rate, differing significantly from the expected 20% (P < .0001; 84% OS at 2 years). CBT with units containing ≥4 × 107 frozen NCs per kilogram is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors. This trial was registered at www.clinicaltrials.gov as #NCT01343953.
AB - Outcomes remain poor for refractory severe aplastic anemia (SAA) patients. Alternative donor transplantation may be considered, but results from previous studies are not encouraging. We conducted a prospective nationwide phase 2 study to assess unrelated cord blood (CB) transplantation (CBT) efficacy and safety in refractory SAA patients (Aplastic Anemia and Cord Blood Transplantation protocol). To demonstrate a significant difference in 1-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients and therefore included 26 (median age, 16 years). Eligibility criteria required 1 or 2 unrelated CB units, containing separately or together >4 × 107 frozen nucleated cells (NCs) per kilogram of recipient body weight. Conditioning regimen comprised fludarabine (FLU), cyclophosphamide (CY), antithymocyte globulin (ATG), and 2-Gy total body irradiation (TBI). With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic graft-versus-host disease were 45.8% and 36%, respectively. Twenty-three patients were alive at 1 year, with an 88.5% overall survival (OS) rate, differing significantly from the expected 20% (P < .0001; 84% OS at 2 years). CBT with units containing ≥4 × 107 frozen NCs per kilogram is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors. This trial was registered at www.clinicaltrials.gov as #NCT01343953.
U2 - 10.1182/blood-2018-01-829630
DO - 10.1182/blood-2018-01-829630
M3 - Article
VL - 132
SP - 750
EP - 754
JO - Blood
JF - Blood
SN - 0006-4971
IS - 7
ER -