Dysplastic neutrophils in the bone marrow of a Shwachman-Diamond syndrome patient

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Allogeneic Bone Marrow Transplantation in Shwachman-Diamond Syndrome with Malignant Myeloid Transformation. A Case Report

Shwachman-Diamond syndrome (SDS) is a rare genetic disorder of unknown pathogenesis involving exocrine pancreatic insufficiency and hematological and skeletal abnormalities. About 25% of patients develop hematopoietic malignancies. We report on a case of acute myeloid leukemia (M2) in a 21-year-old woman affected by SDS. She was treated with conventional chemotherapy (idarubicin plus cytarabine...

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Molecular characterization of a Portuguese patient with Shwachman-Diamond syndrome.

Shwachman-Diamond syndrome (SDS) a rare autosomal recessive disorder described first time 1964 (1), is characterized by the association of exocrine pancreatic and bone marrow dysfunction. Other systemic findings (skeletal, liver and psychomotor) or problems secondary to bone marrow dysfunction may also be detected (1–4). Intermittent or persistent neutropenia is the most common hematologic find...

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Bone Marrow Edema Syndrome (BMES) is an uncommon, self-limited and with unknown etiology characterized by severe pain with no history of trauma. Due to low prevalence of BMES and its non-specific signs, correct diagnosis is often delayed intensifying bone pain and impairing patients’ function and quality of life. In this study we treated a patient with bone marrow edema syndrome in right a...

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A case of Shwachman–Diamond syndrome

Shwachman–Diamond syndrome (SDS) is an autosomal recessive disorder (OMIM 260400), characterized by exocrine pancreatic insufficiency, skeletal abnormalities and bone marrow (BM) dysfunction, with a risk, as high as 30%, to develop myelodysplastic syndrome and/or acute myeloid leukaemia (MDS/AML). The SBDS gene (OMIM 607744) is localized on chromosome 7 at the band q11 and mutations of this gen...

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Defective ribosome assembly in Shwachman-Diamond syndrome.

Shwachman-Diamond syndrome (SDS), a recessive leukemia predisposition disorder characterized by bone marrow failure, exocrine pancreatic insufficiency, skeletal abnormalities and poor growth, is caused by mutations in the highly conserved SBDS gene. Here, we test the hypothesis that defective ribosome biogenesis underlies the pathogenesis of SDS. We create conditional mutants in the essential S...

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ژورنال

عنوان ژورنال: Blood

سال: 2017

ISSN: 0006-4971,1528-0020

DOI: 10.1182/blood-2017-03-773846