Extinguishing the fire in sickle cell anemia

In this issue of Blood, Rees et al(1) report the results of a randomized, double-blind, placebo-controlled, multicenter phase 2a study that tested the safety and efficacy of the interleukin 1-beta (IL-1 beta) neutralizing antibody canakinumab in children and young adults with sickle cell anemia (SCA), chronic pain, and inflammation. The study's primary objective was not met because participants receiving the study drug did not experience a significant reduction in average daily pain, but there was a promising reduction of multiple biomarkers of inflammation, number and duration of hospitalizations, and other patient-reported outcomes of pain and fatigue.

Automated summary

This study tested the safety and efficacy of the IL-1 beta neutralizing antibody canakinumab in children and young adults with sickle cell anemia, chronic pain, and inflammation. While there was no significant reduction in average daily pain, there was a promising reduction in multiple biomarkers of inflammation, number and duration of hospitalizations, and other patient-reported outcomes of pain and fatigue.