Lupus Sci Med. 2026 Mar 18;13(1):e001830. doi: 10.1136/lupus-2025-001830.
ABSTRACT
OBJECTIVES: To define a clinically meaningful treatment response definition for haemolytic anaemia, that will form part of a novel multidomain outcome measure for SLE clinical trials.
METHODS: An international working group comprising 12 clinicians and 4 patients completed a multistep consensus process. First, the scope of haemolytic anaemia for the SLE clinical trial context was defined. Candidate response measures were then nominated via an online survey and shortlisted for evaluation via a systematic literature review (SLR) focussing on relevant measurement properties and clinical trial utility. Informed by the SLR results, the working group used nominal group technique (NGT) to reach consensus on a response measure, as well as entry, clinically meaningful response and complete response thresholds.
RESULTS: The domain scope was unanimously agreed on as 'immune-mediated haemolytic anaemia attributed to active lupus that impacts the patient and is modifiable by therapy to reduce or control disease activity', including Coombs' positive autoimmune haemolytic anaemia and rarer subtypes. Six laboratory measures to fit this scope were shortlisted for the SLR, which screened 1519 articles, of which 10 were included. Consensus via NGT was reached on haemoglobin (Hb) improvement to measure haemolytic anaemia treatment response. Thresholds were defined for entry (Hb <100 g/L with laboratory confirmation of haemolysis), minimum clinically meaningful response (Hb improvement >20 g/L or Hb normalised with ongoing haemolysis) and complete response (Hb normalised without haemolysis).
CONCLUSIONS: Hb, with meaningful thresholds for entry and improvement, will be adopted to classify haemolytic anaemia treatment response in a novel multi-domain outcome measure for SLE clinical trials.
PMID:41850755 | DOI:10.1136/lupus-2025-001830