Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
Biol Blood Marrow Transplant
; 16(2): 263-72, 2010 Feb.
Article
en En
| MEDLINE
| ID: mdl-19822218
ABSTRACT
We conducted a prospective, multicenter investigation of human-leukocyte antigen (HLA) identical sibling bone marrow transplantation (BMT) in children with severe sickle cell disease (SCD) between 1991 and 2000. To determine if children were protected from complications of SCD after successful BMT, we extended our initial study of BMT for SCD to conduct assessments of the central nervous system (CNS) and of pulmonary function 2 or more years after transplantation. In addition, the impact on gonadal function was studied. After BMT, patients with stroke who had stable engraftment of donor cells experienced no subsequent stroke events after BMT, and brain magnetic resonance imaging (MRI) exams demonstrated stable or improved appearance. However, 2 patients with graft rejection had a second stroke after BMT. After transplantation, most patients also had unchanged or improved pulmonary function. Among the 11 patients who had restrictive lung changes at baseline, 5 were improved and 6 had persistent restrictive disease after BMT. Of the 2 patients who had obstructive changes at baseline, 1 improved and 1 had worsened obstructive disease after BMT. There was, however, significant gonadal toxicity after BMT, particularly among female recipients. In summary, individuals who had stable donor engraftment did not experience sickle-related complications after BMT, and were protected from progressive CNS and pulmonary disease.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Enfermedades del Sistema Nervioso Central
/
Estado de Salud
/
Trasplante de Médula Ósea
/
Trastornos Gonadales
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Anemia de Células Falciformes
/
Enfermedades Pulmonares Obstructivas
Tipo de estudio:
Clinical_trials
/
Etiology_studies
/
Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Aspecto:
Determinantes_sociais_saude
/
Patient_preference
Límite:
Adolescent
/
Child
/
Female
/
Humans
/
Male
Idioma:
En
Revista:
Biol Blood Marrow Transplant
Asunto de la revista:
HEMATOLOGIA
/
TRANSPLANTE
Año:
2010
Tipo del documento:
Article
País de afiliación:
Estados Unidos