According to a study published in the American Journal of Hematology, eltrombopag, a thrombopoietin-receptor agonist, increased platelet counts, which translated into significant improvements in patient-reported health-related quality of life (HRQoL) in patients with persistent/chronic immune thrombocytopenia.

The report used the final results of the Eltrombopag Extended Dosing Study (EXTEND; Identifier: NCT00351468) to evaluate changes in patient-reported HRQoL and the association between HRQoL and platelet response. Four HRQoL assessment tools were administered: the Medical Outcome Trust’s Short-Form 36 Health Survey (SF-36v2) with physical (PCS) and mental (MCS) component summaries, the Motivation and Energy Inventory Short Form (MEI-SF), the fatigue subscale of the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue), and the Functional Assessment of Cancer Therapy-Thrombocytopenia tool (FACT-Th6).

Between June 2006 and July 2015, 302 patients participated in EXTEND. The median duration of eltrombopag treatment was 2.4 years (range, 2 days to 8.8 years). All HRQoL tools revealed positive changes from baseline over time. The average time to best improvement among patients ranged from 6 to 10 months, depending on the tool. Median number of days to best improvement for each tool was: 309 for SF-36v2 PCS; 285 for SF-36v2 MCS; 374 for MEI-SF; 276 for FACIT-Fatigue; and 183 for FACT-Th6. The percentage of patients who achieved minimally important differences in HRQoL increases from baseline during treatment for each tool was: 49.5% for SF-36v2 PCS; 50.2% for SF-36v2 MCS; 48.1% for MEI-SF; 64.0% (3 points) and 51/0% (5 points) for FACIT-Fatigue; and 55.0% for FACT-Th6. All 4 HRQoL measurements were positively associated with platelet responses (P <.05 for all except MEI-SF).

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The authors concluded that “while not all patients, [and] not even all responders, have substantial benefit in HRQoL, many individuals had substantial continuing improvements in HRQoL manifest in several instruments” and suggested that future studies on mechanism of improvement in HRQoL should be conducted.


1. Khelif A, Saleh MN, Salama A, et al. Changes in health-related quality of life with long-term eltrombopag treatment in adults with persistent/chronic immune thrombocytopenia: findings from the EXTEND study [published online November 12, 2018]. Am J Hematol. doi:10.1002/ajh.25348