Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children.

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.

[Subsequent malignancies after long-term follow-up of pediatric hematopoietic stem cell transplantation]. / Neoplasias malignas secundarias después de un trasplante de progenitores hematopoyéticos en edad pediátrica.

INTRODUCTION: Survival after hematopoietic stem cell transplantation has improved dramatically in recent years. Unfortunately, there is an increased risk of subsequent malignant neoplasms (SMN) in this population and this represents a significant cause of late mortality. PATIENTS AND METHODS: In this study, we analyzed the incidence of SMN and the associated risk factors in patients referred at a pediatric age for hematopoietic stem cell transplantation (allogeneic or autologous) in our center. RESULTS: We observed 19 cases of SMN in a cohort of 371 patients, with a cumulative incidence of 6, 12, and 36% at 15, 20, and 30 years of follow-up, respectively. The solid tumors were the most prevalent malignancies. The risk was significantly higher than expected in the general population for each tumor type and in the different age ranges (p<.0001). Radiotherapy and chronic GvHD were the main risk factors for the development of SMN in our series. CONCLUSIONS: We observed a high incidence of SMN among hematopoietic stem cell transplantation survivors highlighting the need for life-long surveillance.

Neoplasias malignas secundarias después de un trasplante de progenitores hematopoyéticos en edad pediátrica / Subsequent malignancies after long-term follow-up of pediatric hematopoietic stem cell transplantation

Introducción: La supervivencia después del trasplante de progenitores hematopoyéticos ha mejorado de forma importante en los últimos años. Desafortunadamente, se ha descrito un mayor riesgo de neoplasias malignas secundarias (NMS) en esta población representando una causa importante de mortalidad tardía. Pacientes y métodos: En este estudio, analizamos la incidencia de NMS y los factores de riesgo asociados en los pacientes sometidos en edad pediátrica a un trasplante de progenitores hematopoyéticos (alogénico o autólogo) en nuestro centro. Resultados: Observamos 19 casos de NMS en una cohorte de 371 pacientes, siendo la incidencia acumulada del 6, 12 y 36% a los 15, 20 y 30 años de seguimiento, respectivamente. Los tumores sólidos fueron los más prevalentes. El riesgo fue significativamente mayor de lo esperado en la población general para cada tipo de tumor y en los diferentes rangos de edad (p < 0,0001). La radioterapia y la EICH crónica fueron los principales factores de riesgo para el desarrollo de NMS en nuestra serie. Conclusiones: Hemos observado una alta incidencia de neoplasias malignas secundarias en los supervivientes de un trasplante de progenitores hematopoyéticos evidenciando la necesidad de un seguimiento a largo plazo de esta población de pacientes

Introduction: Survival after hematopoietic stem cell transplantation has improved dramatically in recent years. Unfortunately, there is an increased risk of subsequent malignant neoplasms (SMN) in this population and this represents a significant cause of late mortality. Patients and methods: In this study, we analyzed the incidence of SMN and the associated risk factors in patients referred at a pediatric age for hematopoietic stem cell transplantation (allogeneic or autologous) in our center. Results: We observed 19 cases of SMN in a cohort of 371 patients, with a cumulative incidence of 6, 12, and 36% at 15, 20, and 30 years of follow-up, respectively. The solid tumors were the most prevalent malignancies. The risk was significantly higher than expected in the general population for each tumor type and in the different age ranges (p < .0001). Radiotherapy and chronic GvHD were the main risk factors for the development of SMN in our series. Conclusions: We observed a high incidence of SMN among hematopoietic stem cell transplantation survivors highlighting the need for life-long surveillance