Automated Author Profile

Abdelmoumen, K.

Current S-Index

2.6

Sum of Dataset Indices for all datasets

Average Dataset Index per Dataset

1.3

Average Dataset Index per dataset

Total Datasets

2

Total datasets for this author

Average FAIR Score

51.9%

Average FAIR Score per dataset

Total Citations

0

Total citations to the author's datasets

Total Mentions

0

Total mentions of the author's datasets

S-Index Interpretation

S-Index Over Time

Cumulative Citations Over Time

Cumulative Mentions Over Time

Datasets

Supplementary Material for: Eltrombopag for the Treatment of Severe Inherited Thrombocytopenia

Inherited thrombocytopenias correspond to a group of hereditary disorders characterized by a reduced platelet count, platelet dysfunction, and a family history of thrombocytopenia. It is commonly associated with mucocutaneous bleeding. Thrombocytopenia results from mutations in genes involved in megakaryocyte differentiation, platelet formation, and clearance. Here we report on a patient presenting with severe syndromic inherited thrombocytopenia manifesting as spontaneous mucocutaneous bleeds, requiring frequent platelet transfusions. Thrombocytopenia was explained by the presence of 4 mutations in 3 hematopoietic transcription factor genes: FLI1, RUNX1, and ETV6. The patient was successfully treated with high-dose eltrombopag at 150 mg/day, an orally available non-peptide thrombopoietin receptor agonist. Since the start of treatment 23 months ago, the manifestations of bleeding have resolved, and no platelet transfusions or corticosteroids have been required. The patient has no clinical or laboratory evidence of myeloid malignancy so far.

Authors

  • Abdelmoumen, K. ;
  • Fabre, M. ;
  • Ducastelle-Lepretre, S. ;
  • Favier, R. ;
  • Ballerini, P. ;
  • Bordet, J.C. ;
  • Dargaud, Y.
0 Citations0 Mentions48% FAIR1.2 Dataset Index
10.6084/m9.figshare.13013669January 2020

Supplementary Material for: Eltrombopag for the Treatment of Severe Inherited Thrombocytopenia

Inherited thrombocytopenias correspond to a group of hereditary disorders characterized by a reduced platelet count, platelet dysfunction, and a family history of thrombocytopenia. It is commonly associated with mucocutaneous bleeding. Thrombocytopenia results from mutations in genes involved in megakaryocyte differentiation, platelet formation, and clearance. Here we report on a patient presenting with severe syndromic inherited thrombocytopenia manifesting as spontaneous mucocutaneous bleeds, requiring frequent platelet transfusions. Thrombocytopenia was explained by the presence of 4 mutations in 3 hematopoietic transcription factor genes: FLI1, RUNX1, and ETV6. The patient was successfully treated with high-dose eltrombopag at 150 mg/day, an orally available non-peptide thrombopoietin receptor agonist. Since the start of treatment 23 months ago, the manifestations of bleeding have resolved, and no platelet transfusions or corticosteroids have been required. The patient has no clinical or laboratory evidence of myeloid malignancy so far.

Authors

  • Abdelmoumen, K. ;
  • Fabre, M. ;
  • Ducastelle-Lepretre, S. ;
  • Favier, R. ;
  • Ballerini, P. ;
  • Bordet, J.C. ;
  • Dargaud, Y.
0 Citations0 Mentions56% FAIR1.4 Dataset Index
10.6084/m9.figshare.13013669.v1January 2020