Allogeneic stem cell transplantation for the treatment of refractory scleromyxedema.
Transl Res
; 165(2): 321-4, 2015 Feb.
Article
en En
| MEDLINE
| ID: mdl-24999269
ABSTRACT
Scleromyxedema is a rare disorder of connective tissue with unknown etiology. Its manifestation includes a generalized mucin deposition, which is frequently associated with paraproteinemia. The course of scleromyxedema is progressive and often lethal. As a result of its poorly understood pathogenesis, there is no causative treatment option. The efficacy of cytoreductive agents and autologous stem cell transplantation has been reported, but so far allografting as a treatment option has not yet been documented. Herein, we report on a patient with severe neurologic involvement and refractory course attaining durable remission after receiving an allogeneic hematopoietic cell transplant from an human leukocyte antigen-matched sibling. This case not only illustrates a potential new treatment option for selected patients, but also provides insights into the pathogenesis of this rare disease.
Texto completo:
1
Colección:
01-internacional
Asunto principal:
Trasplante de Células Madre Hematopoyéticas
/
Escleromixedema
Tipo de estudio:
Etiology_studies
Límite:
Adult
/
Humans
/
Male
Idioma:
En
Revista:
Transl Res
Asunto de la revista:
MEDICINA
/
TECNICAS E PROCEDIMENTOS DE LABORATORIO
Año:
2015
Tipo del documento:
Article