Eltrombopag use in a patient with Wiskott–Aldrich syndrome

@article{Gabelli2017EltrombopagUI,
  title={Eltrombopag use in a patient with Wiskott–Aldrich syndrome},
  author={Maria Gabelli and Antonio Marzollo and Lucia Dora Notarangelo and Giuseppe Basso and Maria Caterina Putti},
  journal={Pediatric Blood \& Cancer},
  year={2017},
  volume={64}
}
Wiskott–Aldrich syndrome (WAS) is an inherited X‐linked disorder characterized by microthrombocytopenia, immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice. Eltrombopag, a thrombopoietin receptor agonist, may be useful to prevent bleeding while awaiting HSCT. We present a case of a male with WAS, profound thrombocytopenia, and bleeding diathesis successfully managed with eltrombopag before HSCT. Eltrombopag was given for 32 weeks obtaining a… 
Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome
  • F. Candotti
  • Medicine, Biology
    Journal of Clinical Immunology
  • 2017
TLDR
The Wiskott-Aldrich syndrome has a wide clinical spectrum ranging from mild, isolated thrombocytopenia to full-blown presentation that can be complicated by life-threatening hemorrhages, immunodeficiency, atopy, autoimmunity, and cancer.
Efficacy of romiplostim in treatment of thrombocytopenia in children with Wiskott–Aldrich syndrome
TLDR
It is demonstrated that romiplostim can be used to increase platelet counts and reduce the risks of life‐threatening bleeding in WAS patients awaiting haematopoietic stem cell transplantation or forgoing the procedure for various reasons.
Eltrombopag for the Treatment of Severe Inherited Thrombocytopenia
TLDR
A patient presenting with severe syndromic inherited thrombocytopenia manifesting as spontaneous mucocutaneous bleeds, requiring frequent platelet transfusions, was successfully treated with high-dose eltrombopag at 150 mg/day, an orally available non-peptide throm bopoietin receptor agonist.
Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott–Aldrich Syndrome
TLDR
Wiskott–Aldrich syndrome is a rare X-linked recessive inborn error of immunity first described in 1937, characterized by the triad of thrombocytopenia with small platelets, recurrent infections due to combined immunodeficiency, and eczema.
Report of clinical presentations and two novel mutations in patients with Wiskott-Aldrich syndrome/X-linked Thrombocytopenia
TLDR
Two patients with Wiskott-Aldrich syndrome/X-linked thrombocytopenia had severe clinical phenotypes compatible with classic WAS and developed lethal outcomes with intracranial hemorrhage and one patient with XLT developed pineoblastoma.
Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial
TLDR
In conclusion, eltrombopag was safe and effective in increasing platelet count and reducing bleeding symptoms in different forms of inherited thrombocytopenia.
Platelets in Wiskott‐Aldrich syndrome: Victims or executioners?
TLDR
An overview of the state‐of‐the art platelets as immune modulators at the interface between hemostasis and the immune system, which suggests that these cells may have a direct role in the pathogenesis of immune dysregulation in WAS.
Role of Thrombopoietin Receptor Agonists in Inherited Thrombocytopenia
TLDR
An updated and comprehensive review about the efficacy and safety of TPO-RA in ITs is carried out.
Thrombopoietin receptor agonists in hereditary thrombocytopenias
TLDR
It is believed that TPO‐RAs will play a major role in the treatment of HTPs, particularly myosin heavy chain 9‐related disease, Wiskott–Aldrich syndrome, X‐linked thrombocytopenia, and THPO mutations, and their use in patients with these disorders is discussed.
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