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Inicio Medicina Clínica (English Edition) Hematopoietic stem cell transplantation in pediatric patients with β-thalassemi...
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Vol. 152. Issue 4.
Pages 135-140 (February 2019)
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Vol. 152. Issue 4.
Pages 135-140 (February 2019)
Original article
Hematopoietic stem cell transplantation in pediatric patients with β-thalassemia and sickle cell disease: An experience of the Spanish Working Group for Bone Marrow Transplantation in Children (GETMON)
Trasplante de progenitores hematopoyéticos en niños con β-talasemia y enfermedad drepanocítica: experiencia del grupo GETMON
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Laura Alonsoa,
Corresponding author
l.alonso@vhebron.net

Corresponding author.
, Marta González-Vicentb, Cristina Belendezc, Isabel Badelld, Ana Sastree, Antonia Rodríguez-Villaf, Mar Bermúdez-Cortésg, Raquel Hladuna, Cristina Díaz de Herediaa
a Unidad Pediátrica de Trasplante Hematopoyético, Hospital Universitario Vall d’Hebron, Barcelona, Spain
b Unidad Pediátrica de Trasplante Hematopoyético, Hospital Niño Jesús, Madrid, Spain
c Unidad Pediátrica de Trasplante Hematopoyético, Hospital Universitario Gregorio Marañón, Madrid, Spain
d Unidad Pediátrica de Trasplante Hematopoyético, Hospital de la Santa Creu i Sant Pau, Universidad Autónoma, Barcelona, Spain
e Unidad Pediátrica de Trasplante Hematopoyético, Hospital La Paz, Madrid, Spain
f Unidad Pediátrica de Trasplante Hematopoyético, Hospital Universitario Reina Sofía, Córdoba, Spain
g Unidad Pediátrica de Trasplante Hematopoyético, Hospital Universitario Virgen de la Arrixaca, Murcia, Spain
Article information
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Table 1. Patients with haemoglobinopathies who received a haematopoietic stem cell transplant.
Abstract
Background and objectives

A recently occurring increase of the prevalence of haemoglobinopathies, β-thalassaemia major (TM) and sickle cell disease (SCD) over the last two decades in our country has generated new needs in terms of medical resources for both prevention and treatment of these patients. Allogeneic haematopoietic stem cell transplant (allo-HSCT) is a curative treatment available for patients who have severe haemoglobinopathies. The main objective of this study was to evaluate the results of allo-HSCT in paediatric patients with TM or SCD performed in paediatric haematopoietic transplant units within the Spanish Group of Bone Marrow Transplantation in Children (GETMON).

Material and methods

Retrospective review of patients undergoing HSCT in the GETMON units until 2015.

Results

A total of 65 patients were analysed (43 patients were affected with TM and 22 with SCD), who received allo-HSCT in 6 GETMON units between November 1989 and December 2014. Event-free survival three years post-transplant was 81% and overall survival 92% in patients with TM. Event-free survival three years post-transplant was 79% and overall survival 85% in patients with SCD.

Conclusions

The results of this series are comparable to the results of other international series and offer a platform from which to continue trying to improve the evolution of these patients.

Keywords:
Thalassaemia
Sickle cell disease
Bone marrow transplant
Haematopoietic stem cell transplantation
Resumen
Antecedentes y objetivos

El incremento descrito en la prevalencia de hemoglobinopatías, de β-talasemia mayor (TM) y de enfermedad drepanocítica (ED) que ha ocurrido en las últimas dos décadas en nuestro país ha generado nuevas necesidades en cuanto a recursos médicos tanto para la prevención como para el tratamiento de estos pacientes. El trasplante alogénico de progenitores hematopoyéticos (alo-TPH) es el tratamiento curativo disponible en nuestro medio para pacientes con hemoglobinopatías graves. El objetivo principal de este estudio fue conocer los resultados del alo-TPH en pacientes pediátricos con TM o ED realizados en unidades de trasplante hematopoyético pediátrico incluidas dentro del Grupo Español de Trasplante de Médula Ósea en Niños (GETMON).

Material y métodos

Revisión retrospectiva de los pacientes sometidos a TPH en unidades de TPH del GETMON hasta el año 2015.

Resultados

Se analizaron un total de 65 pacientes (43 pacientes afectados de TM y 22 de ED) que recibieron el alo-TPH en 6 unidades GETMON entre noviembre de 1989 y diciembre de 2014. La supervivencia libre de eventos 3 años postrasplante fue del 81% y la supervivencia global del 92% en pacientes con TM. La supervivencia libre de eventos 3 años postrasplante fue del 79% y la supervivencia global del 85% en pacientes con ED.

Conclusiones

Los resultados de esta serie son comparables a los resultados de otras series internacionales y ofrecen un punto de partida para continuar intentando mejorar la evolución de estos pacientes.

Palabras clave:
Talasemia
Drepanocitosis
Trasplante de médula ósea
Trasplante de progenitores

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