Correction of Severe Myelofibrosis, Impaired Platelet Functions and Abnormalities in a Patient with Gray Platelet Syndrome Successfully Treated by Stem Cell Transplantation.
Platelets
; 31(4): 536-540, 2020 May 18.
Article
en En
| MEDLINE
| ID: mdl-31502501
ABSTRACT
Gray platelet syndrome (GPS) is an inherited disorder. Patients harboring GPS have thrombocytopenia with large platelets lacking α-granules. A long-term complication is myelofibrosis with pancytopenia. Hematopoietic stem cell transplant (HSCT) could be a curative treatment. We report a male GPS patient with severe pancytopenia, splenomegaly and a secondary myelofibrosis needing red blood cells transfusion. He received an HSCT from a 10/10 matched HLA-unrelated donor after a myeloablative conditioning regimen. Transfusion independence occurred at day+21, with a documented neutrophil engraftment. At day+ 180, we added ruxolitinib to cyclosporine and steroids for a moderate chronic graft versus host disease (GVHD) and persistent splenomegaly. At day+240 GVHD was controlled and splenomegaly reduced. Complete donor chimesrism was documented in blood and marrow and platelets functions and morphology normalized. At day+ 720, the spleen size normalized and there was no evidence of marrow fibrosis on the biopsy. In GPS, HSCT may be a curative treatment in selected patients with pancytopenia and myelofibrosis.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Plaquetas
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Trasplante de Células Madre Hematopoyéticas
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Síndrome de Plaquetas Grises
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Mielofibrosis Primaria
Tipo de estudio:
Etiology_studies
Límite:
Adult
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Humans
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Male
Idioma:
En
Año:
2020
Tipo del documento:
Article