High dose romiplostim shows promise in patients of refractive aplastic anaemia

On data analysis, the following facts emerged.

• The primary efficacy endpoint of the proportion of patients achieving any haematological (platelet, neutrophil and erythrocyte) response at week 27 was 84% [95% confidence interval (CI) 66–95%].
• Trilineage response was 39% (95% CI 22–58%) at week 53. The most common treatment‐related adverse events (AEs) were headache and muscle spasms (each 13%).
• All AEs were mild or moderate except for three patients with Grade 3 hepatic AEs; no AEs necessitated romiplostim discontinuation.
• Two patients developed cytogenetic abnormalities, of whom one returned to normal karyotype at last follow‐up.

"A limitation of the present study was the recruitment of a relatively small number of patients, as well as the inclusion of a somewhat heterogeneous patient population with respect to their disease severity. However, recruitment of patients with refractory AA is restricted given the rarity of the condition. Long‐term follow‐up data will be required to confirm the durability of response and monitor for potential clonal evolution." wrote the team.

Primary source: British Journal of Haematology

For the full article follow the link: https://doi.org/10.1111/bjh.17190