Brief clinical and laboratory observationCongenital agranulocytosis terminating in acute myelomonocytic leukemia1
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Cited by (73)
Neonatal manifestations of inherited bone marrow failure syndromes
2016, Seminars in Fetal and Neonatal MedicineCitation Excerpt :G-CSF is a mainstay of treatment of, or prophylaxis from, life-threatening bacterial infections. Patients with SCN were reported to be at increased risk of MDS and AML prior to the availability of G-CSF treatment and now G-CSF appears to add to that risk [69–72]. Notably, a prospective study on 374 patients on G-CSF treatment and overall risk of MDS/AML was 15–25% by age 15 years.
Epidemiology of Congenital Neutropenia
2013, Hematology/Oncology Clinics of North AmericaCitation Excerpt :After the efficacy of growth factors in neutropenia associated with chemotherapy was demonstrated46 and the need for long-term administration in some cases emerged,47 questions about their safety were raised, especially regarding the risk of malignant transformation. The issue was mentioned in the first article reporting the effect of GCSF in this setting,46 particularly because leukemia had been observed in patients with congenital neutropenia who survived beyond their first decade of life.48–50 At that time, the knowledge about congenital neutropenia was limited to phenotype, with large categories, such as Kostmann disease, Shwachman-Diamond disease, cyclic neutropenia, and idiopathic neutropenia.
G-CSF and its receptor in myeloid malignancy
2010, BloodCongenital Neutropenia Syndromes
2008, Immunology and Allergy Clinics of North AmericaHematopoietic Growth Factors for the Treatment of Inherited Cytopenias
2007, Seminars in HematologyCitation Excerpt :For those patients who do not respond to G-CSF treatment alone or in combination with SCF, hematopoietic stem cell transplantation (HSCT) is the only currently available treatment19,22; when successful, hematopoiesis normalizes and no cytokine treatment is required. Leukemic transformation has been reported in single patients with CN.23,24 The first 374 patients (1987–2000) with CN on long-term G-CSF who were enrolled in the SCNIR have been studied in detail to identify risks for leukemic transformation.25
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Supported in part by the Stella and A1 Dershewitz Research Fund.