Trasplante haploidéntico de células progenitoras hematopoyéticas con ciclofosfamida después del trasplante en errores innatos de la inmunidad: experiencia en un centro de referencia en Colombia
Resumen
Introducción. Los errores innatos de la inmunidad son enfermedades raras causadas por más de 400 mutaciones genéticas que afectan al sistema inmunológico y aumentan la susceptibilidad a infecciones, la autoinmunidad y las neoplasias malignas. El trasplante de progenitores hematopoyéticos es una opción curativa para algunos errores innatos de la inmunidad y, específicamente, el trasplante haploidéntico es una alternativa viable en ausencia de un donante idéntico.
Objetivo. Determinar la supervivencia, utilidad del monitoreo semanal del quimerismo, reconstitución inmunológica y complicaciones de los pacientes con errores innatos de la inmunidad sometidos a trasplante haploidéntico de progenitores hematopoyéticos en un centro de referencia en Colombia.
Materiales y métodos. Se realizó un estudio observacional, retrospectivo, de serie de casos, con pacientes pediátricos sometidos a trasplante haploidéntico de progenitores hematopoyéticos, usando ciclofosfamida después del trasplante y haciendo seguimiento semanal de quimerismo. Se analizó la supervivencia con el método de Kaplan-Meier.
Resultados. Se incluyeron 16 pacientes con trasplante de donante familiar haploidéntico. El diagnóstico más frecuente fue inmunodeficiencia combinada grave (n = 5). Once de diecisiete recibieron un esquema de acondicionamiento no mieloablativo. La enfermedad aguda de injerto contra huésped se desarrolló en 12 de 16 pacientes y en 3 fue de grado III-IV. Las infecciones posteriores al trasplante afectaron a 14 pacientes, con predominio de agentes bacterianos. La mediana de quimerismo de células T fue mayor al 80 % durante el seguimiento. La reconstitución de linfocitos B y T se logró en más de 11 pacientes La supervivencia global a cinco años fue del 81 % y, a 100 días, fue del 91 %.
Conclusión. El trasplante haploidéntico de progenitores hematopoyéticos utilizando ciclofosfamida postrasplante, es una alternativa factible en pacientes con errores innatos de la inmunidad en ausencia de un donante idéntico. El monitoreo seriado del quimerismo es útil para el seguimiento del injerto.
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