Trasplante de médula ósea en pacientes con anemia falciforme. Experiencia en un centro / Bone marrow transplant in patients with sickle cell anaemia. Experience in one centre

RESUMEN

INTRODUCCIÓN: La enfermedad de células falciformes (ECF), pese a la mejora en el manejo médico, persiste asociada a morbilidad y a menor supervivencia. El alotrasplante de progenitores hematopoyéticos (alo-TPH) es actualmente la única opción curativa. Describir la evolución clínico-analítica de los pacientes trasplantados en nuestro centro. MATERIAL Y MÉTODO: Estudio unicéntrico descriptivo, incluye a pacientes con ECF en los que se realiza alo-TPH de médula ósea de hermano HLA-idéntico desde enero del 2010 hasta diciembre del 2014. Se recogen datos epidemiológicos, clínicos y analíticos con tiempo de seguimiento hasta diciembre del 2015. Los datos se presentan como frecuencias, porcentajes y medianas (rango). RESULTADOS: Se recluta a 11 pacientes (8 varones), mediana de edad 7 años (2-13), todos ellos con comorbilidad previa al TPH. Se consigue injerto estable en 10/11 pacientes, quimerismo completo en 9/11 y quimerismo mixto estable tras un año del TPH en 1/11. Un paciente presenta fallo secundario de injerto con reaparición de clínica el día +180. Complicaciones post-TPH complicaciones neurológicas 4/11 pacientes (hemorragia subaracnoidea, crisis), HTA 7/11, fallo renal agudo 3/11, reactivación CMV 9/11, EICHa cutáneo 6/11, uno de ellos desarrolla EICH intestinal grado IV causando su fallecimiento (día +51). Ningún paciente desarrolla EICH crónico. Supervivencia global y libre de eventos a los 3,1 años de seguimiento 90,9 y 81,9%, respectivamente. CONCLUSIONES: El alo-TPH, única opción curativa, no está exento de morbimortalidad, encontramos un riesgo de muerte similar a otras series (1/11), siendo su primera causa el EICH agudo. Otros problemas son fallo de injerto (1/11) y complicaciones neurológicas (4/11), aunque las secuelas permanentes son leves

ABSTRACT

INTRODUCTION: Sickle cell disease (SCD), despite the improvement in the medical management, is still associated with severe morbidity and decreased survival. Allogenic hematopoietic stem cell transplantation (Allo-HSCT) currently provides the only curative therapy. A report is presented on our experience in children with SCD, who underwent Allo-HSCT in a single centre. Material and method. A single centre descriptive study was conducted on patients with SCD who underwent a bone marrow transplant from an HLA-identical sibling donor between January 2010 and December 2014. Epidemiological, clinical and analytical parameters were collected with a follow-up to December 2015. Data are presented as frequencies, percentages, and medians (range). RESULTS: Allo-HCST was performed in 11 patients (8 males) with a median age of 7 years (2-13), all of them with comorbidity prior to the HCST. A stable graft was achieved in 10 out of 11 patients, 9 of them with complete donor chimerism, and one patient with stable mixed chimerism after 1 year of allo-HSCT. One patient has secondary graft failure with re-appearance of symptoms associated with SCD on day 180. Complications of Allo-HSCT are arterial hypertension 7/11, acute renal failure 3/11, CMV reactivation 9/11, neurological complications 4/11 (subarachnoid haemorrhage, seizure), and acute graft versus host disease (aGVHD) of the skin 6/11, one of whom developed grade IV intestinal aGVHD, causing his death (day 51). None of the patients developed chronic GVHD. The overall survival and event-free survival was 90.9% and 81.9%, respectively, with a median follow-up of 3.1 (1-5.7) years. CONCLUSIONS: Allo-HSCT, the only curative therapy, remains associated with morbidity. There was a transplant related mortality in our study, consistent with multicentre studies (1/11), and with aGVHD being the main cause. Other problems still include graft failure (1/11), and neurological complications (4/11), although the permanent sequelae are mild