PF349 57 PATIENTS WITH HAX1 ASSOCIATED CONGENITAL NEUTROPENIA: AN ANALYSIS OF THE EUROPEAN SEVERE CHRONIC NEUTROPENIA INTERNATIONAL REGISTRY (SCNIR)

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HAX1 mutation in an infant with severe congenital neutropenia.

Severe congenital neutropenia (SCN) is a rare primary myelopoiesis disorder, characterized by persistent severe neutropenia and early-onset bacterial infections. Herein, we describe an 11-month-old male who was referred with recurrent cutaneous infections and chronic diarrhea. Serial complete blood counts indicated persistent neutropenia. Bone marrow aspiration of the patient demonstrated matur...

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Novel HAX1 gene mutations associated to neurodevelopment abnormalities in two Italian patients with severe congenital neutropenia.

We read with interest the recent perspective article by Klein. 1 Genetic analysis in individuals with severe congenital neutropenia (SCN) indicates that 60% of cases were attributable to heterozygous mutation in ELA2 gene encoding neutrophil elastase. 2 Homozygous mutation in HAX1 gene has been identified in patients with autosomal recessive form of SCN (Kostmann syndrome). 3 Patients with ELA2...

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Outcome and management of pregnancies in severe chronic neutropenia patients by the European Branch of the Severe Chronic Neutropenia International Registry.

Long-term granulocyte-colony stimulating factor treatment has been shown to be safe and effective in severe chronic neutropenia patients. However, data on its use during pregnancy are limited. To address this issue, we analyzed all pregnancies reported to the European branch of the Severe Chronic Neutropenia International Registry since 1994. A total of 38 pregnancies in 21 women with chronic n...

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Severe congenital neutropenia in 2 siblings of consanguineous parents. The role of HAX1 deficiency.

Severe congenital neutropenia (SCN) is a rare primary immunodeficiency disease that is characterized by persistent severe neutropenia and severe early-onset bacterial infections. We report the case of 2 siblings with SCN who were the children of consanguineous parents. The HAX1 mutation was identified in both siblings. Both patients suffered from oral ulcers, candidiasis, respiratory tract infe...

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Severe congenital neutropenia: case report

Introduction Neutropenia is defined in the literature as absolute neutrophil counts in peripheral blood of less than 1500 cells/mm3 in more than one year old and less than 2000cells/mm3 in children in the first year old of life. Neutropenia is classified as mild, moderate or severe, and may be congenital or acquired, persistent or not. Kostmann syndrome is a severe neutropenia, the incidence va...

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ژورنال

عنوان ژورنال: HemaSphere

سال: 2019

ISSN: 2572-9241

DOI: 10.1097/01.hs9.0000559608.06488.2b