EFFECT OF IANALUMAB PLUS ELTROMBOPAG ON PATIENT-REPORTED OUTCOMES IN PRIMARY IMMUNE THROMBOCYTOPENIA: RESULTS FROM THE VAYHIT2 PHASE 3 TRIAL
Nichola Cooper et al
Imperial College London, Centre for Haematology, Department of Immunology and Inflammation, London, United Kingdom
Background
- In the 2018 immune thrombocytopenia (ITP) World Impact Survey, between one-third and one-half of patients reported a meaningful impact on their health-related quality of life (HRQoL), including fatigue, reduced ability to exercise, and high symptom burden.1
- The phase 3 VAYHIT2 trial (NCT05653219) demonstrated prolonged time to treatment failure and improved stable response rates at 6 months with ianalumab plus eltrombopag vs placebo plus eltrombopag in patients with ITP who had insufficient response to or relapsed after first-line corticosteroids.
- In addition, improvement in fatigue was observed using the Patient-Reported Outcomes Measurement Information System Short Form v1.0 Fatigue 13a.2
- The aim of the study is to further characterize the impact of ianalumab plus eltrombopag on patient-reported outcomes (PROs) using a disease-specific instrument validated to measure the burden of ITP symptoms and its treatment.
Methods
- Patients (N=152) were randomized (1:1:1) to receive ianalumab 9 or 3 mg/kg or placebo, plus eltrombopag. Ianalumab or placebo was administered as four once-monthly intravenous infusions, alongside daily eltrombopag for 16 weeks.
- This was followed by an 8-week eltrombopag tapering period until discontinuation in eligible patients by week 25. PROs were collected at on-treatment visits and during post-treatment follow-up until end of study and assessed using the Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ), a 44-item, 10-scale instrument for HRQoL in adults with ITP. Each scale was scored from 0 to100, with higher scores indicating better HRQoL.
- Scores were analyzed using repeated linear mixed models, with least-square (LS) mean changes from baseline calculated for each treatment group. This analysis focused on the ITP-PAQ scales of Symptoms, Fatigue, Bother, and Activity, as these were secondary endpoints of interest.
Results
- At data cutoff (June 20, 2025), the median duration of follow-up was 12.9, 13.6, and 11.6 months with ianalumab 9 mg/kg plus eltrombopag (hereafter referred to as the ianalumab 9 mg/kg group), ianalumab 3 mg/kg plus eltrombopag (ianalumab 3 mg/kg group), and placebo plus eltrombopag (placebo group), respectively.
- Across ITP-PAQ scales of Symptoms, Fatigue, Bother, and Activity, scores at most time points were highest in the ianalumab 9 mg/kg group (Figure); however, improvements were observed in all treatment groups, beginning early in the treatment period and extending through eltrombopag tapering and discontinuation (week 25), with LS-mean (95% confidence interval) changes from baseline for ITP-PAQ Symptoms being 21.6 (17.6‐25.7) and 18.4 (14.2‐22.5) with ianalumab 9 and 3 mg/kg, respectively, and 16.7 (12.8‐20.6) with placebo; for Fatigue, these figures were 21.2 (15.3‐27.2), 12.9 (6.6‐19.2), and 13.6 (7.7‐19.6).
- Corresponding improvements from baseline at week 25 for Bother were 26.4 (20.9‐31.8), 25.4 (19.7‐31.2), and 19.0 (13.5‐24.4); and 25.0 (18.0‐32.1), 22.8 (15.3‐30.3), and 20.5 (13.4‐27.5) for Activity.
Conclusion
- Improvements in the ITP-specific PROs across the Symptoms, Fatigue, Bother, and Activity scales were observed in all groups, with greater and more consistent improvements observed in the ianalumab 9 mg/kg group. T
- There was no indication of increased treatment-related bother or reduced HRQoL due to the addition of ianalumab.
References
- Cooper N, et al. Am J Hematol. 2021;96:188–198.
- Cuker A, et al. N Engl J Med. 2025. doi:10.1056/NEJMoa2515168.
