Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children
| Autor: | José Luis Fuster, J. M. Perez-Hurtado, Monserrat Torrent, Laura C. Alonso, Elena Cela, Cristina Beléndez, Grupo Español de Trasplante de Médula Ósea en Niños, Cristina Díaz-de-Heredia, Marta González-Vicent, María Isabel Benítez-Carabante, María Luz Uría-Oficialdegui |
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| Rok vydání: | 2020 |
| Předmět: |
Male
medicine.medical_specialty Transplantation Conditioning Cyclophosphamide medicine.medical_treatment Graft vs Host Disease Hematopoietic stem cell transplantation Anemia Sickle Cell Severity of Illness Index 03 medical and health sciences 0302 clinical medicine Internal medicine Clinical endpoint medicine Humans Transplantation Homologous Cumulative incidence Child Retrospective Studies business.industry Incidence Siblings Hematopoietic Stem Cell Transplantation Retrospective cohort study Hematology General Medicine Transplantation surgical procedures operative medicine.anatomical_structure Spain 030220 oncology & carcinogenesis Female Bone marrow business Busulfan 030215 immunology medicine.drug |
| Zdroj: | European journal of haematologyREFERENCES. 106(3) |
| ISSN: | 1600-0609 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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