Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children
Por:
Benitez-Carabante, MI, Belendez, C, Gonzalez-Vicent, M, Alonso, L, Uria-Oficialdegui, ML, Torrent, M, Perez-Hurtado, JM, Fuster, JL, Cela, E, Diaz-de-Heredia, C
Publicada:
1 mar 2021
Ahead of Print:
1 ene 2021
Resumen:
Objectives The prevalence of sickle cell disease (SCD) in Spain is markedly inferior compared with other European and Mediterranean countries. However, the diagnosis of new patients with SCD is expected to increase. In this multicenter retrospective study, we analyze the hematopoietic stem cell transplantation (HSCT) results obtained in Spain.
Methods Forty-five patients who underwent a matched sibling donor (MSD) HSCT between 1999 and 2018 were included. Primary endpoint was event-free survival (EFS), and secondary endpoints included acute and chronic graft-versus-host disease (GvHD) and overall survival (OS).
Results Bone marrow was the most frequent stem cell source (93.3%). Most patients received a conditioning regimen based on busulfan and cyclophosphamide (69%). Cumulative incidence of grade III-IV acute GvHD and chronic GvHD was 6.8% (95% CI: 2.3%-20.1%) and 5.4% (95% CI: 1.38%-19.9%), respectively. EFS and overall survival (OS) at 3 years post-HSCT were 89.4% (95% CI: 73.9%-95.9%) and 92.1% (95% CI: 77.2%-97.4%), respectively. All patients aged <= 5 presented 100% EFS and OS.
Conclusions An early referral to HSCT centers should be proposed early in life, before severe complications occur. MSD HSCT should be considered a curative option for all patients aged <= 5 years and for older pediatric patients who present complications derived from the disease.
Filiaciones:
Benitez-Carabante, MI:
Vall dHebron Inst Recerca VHIR, Hosp Univ Vall dHebron, Dept Pediat Hematol & Oncol, Barcelona, Spain
Belendez, C:
Hosp Gen Univ Gregorio Maranon, Dept Pediat Hematol & Oncol, Madrid, Spain
Gonzalez-Vicent, M:
Hosp Infantil Univ Nino Jesus, Dept Pediat Hematol & Oncol, Madrid, Spain
Alonso, L:
Vall dHebron Inst Recerca VHIR, Hosp Univ Vall dHebron, Dept Pediat Hematol & Oncol, Barcelona, Spain
Uria-Oficialdegui, ML:
Vall dHebron Inst Recerca VHIR, Hosp Univ Vall dHebron, Dept Pediat Hematol & Oncol, Barcelona, Spain
Torrent, M:
Hosp Santa Creu & Sant Pau, Dept Pediat Hematol & Oncol, Barcelona, Spain
Perez-Hurtado, JM:
Hosp Univ Virgen Rocio, Dept Hematol, Seville, Spain
Fuster, JL:
Hosp Clin Univ Virgen Arrixaca, Dept Pediat Hematol & Oncol, Murcia, Spain
Cela, E:
Hosp Gen Univ Gregorio Maranon, Dept Pediat Hematol & Oncol, Madrid, Spain
Diaz-de-Heredia, C:
Vall dHebron Inst Recerca VHIR, Hosp Univ Vall dHebron, Dept Pediat Hematol & Oncol, Barcelona, Spain
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