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Background: Oral thrombopoietin receptor agonists are used to treat thrombocytopenia in patients with chronic liver disease who are scheduled for invasive procedures. The efficacy of lusutrombopag based on the pretreatment platelet count was investigated.
Methods: Patients treated at nine hospitals from December 2015 to December 2023 were included.
Treatment of refractory/relapsed Diamond-Blackfan anaemia with eltrombopag.
Br J Haematol
May 2024
Translational Stem Cell Biology Branch, NHLBI, Bethesda, Maryland, USA.
Article Synopsis
- Diamond-Blackfan anaemia (DBA) is a rare genetic condition that leads to bone marrow failure, primarily due to problems with ribosomal function, resulting in issues with red blood cell (RBC) production and excess reactive iron.
- A pilot study tested the effectiveness of eltrombopag, a drug that acts as an iron chelator, in improving RBC production among DBA patients, with some positive results but limited overall response rates.
- While eltrombopag showed promise in increasing hemoglobin levels and reducing blood transfusion needs in one patient, complications like thrombocytosis led to dose adjustments or discontinuation in a significant portion of participants, highlighting challenges in treatment and the need for further research.
Efficacy and safety of high dose recombinant human thrombopoietin in the treatment of immune thrombocytopenia.
Platelets
December 2023
Department of Hematology, The Second Affiliated Hospital of Kunming Medical University, Kunming, China.
The conventional dose of recombinant human thrombopoietin (rhTPO) in the treatment of immune thrombocytopenia (ITP) is 300 U/kg per day, but the clinical reaction rate is not satisfactory. Accordingly, we explored the efficacy and safety of increasing rhTPO dose in the treatment of ITP. A retrospective study was conducted to collect the clinical data of 105 ITP patients who were divided into two groups, a low-dose group (15 000 U/day) and a high-dose group (30 000 U/day) according to the dose of rhTPO.
View Article and Find Full Text PDFTwo Refractory Immune Thrombocytopenia Case Reports Showing Responsiveness to Fostamatinib.
Case Rep Hematol
June 2023
U.O.C di Ematologia, Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione, ARNAS, Garibaldi di Catania, Catania, Italy.
Immune thrombocytopenia (ITP) is immune-mediated platelet loss due to increased destruction and insufficient production. Treatment guidelines provide for first-line steroid-based therapies followed by thrombopoietin receptor agonists (TPO-RAs) and fostamatinib for chronic ITP. Fostamatinib demonstrated efficacy in phase 3 FIT trials (FIT1 and FIT2) mainly in second-line therapy resulting in the maintenance of stable platelet values.
View Article and Find Full Text PDFKidney-Derived Thrombopoietin and Platelet Generation-A Proinflammatory Loop in CKD.
J Am Soc Nephrol
July 2023
Centre for Inflammatory Diseases, Monash University Department of Medicine, Monash Medical Centre, Clayton, Victoria, Australia.
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