Disease-modifying treatments for primary autoimmune haemolytic anaemia

Liu, Anthony Pak-Yin; Cheuk, Dkl

doi:10.1002/14651858.cd012493.pub2

Cited by 2 publications

(1 citation statement)

References 54 publications

(11 reference statements)

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“…Results from various studies have been published 2,26 . It is important to recognize that most trials reported data based on heterogeneous outcome measures, which make the trials difficult to compare.…”

Section: Discussionmentioning

confidence: 99%

Fostamatinib for warm antibody autoimmune hemolytic anemia: Phase 3, randomized, double‐blind, placebo‐controlled, global study (FORWARD)

Kuter,

Piatek,

Röth

et al. 2023

American J Hematol

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Warm antibody autoimmune hemolytic anemia (wAIHA) is characterized by hemolysis and symptomatic anemia with no approved treatment options. Fostamatinib is an oral spleen tyrosine kinase inhibitor approved in the US and Europe for treatment of adults with chronic immune thrombocytopenia. In this phase 3 study, patients with an insufficient response to ≥1 prior wAIHA treatment were randomized to fostamatinib or placebo. The primary endpoint was the proportion of patients to achieve a durable hemoglobin (Hgb) response (Hgb ≥10 g/dL and increase from baseline of ≥2 g/dL on 3 consecutive visits) during the 24‐week treatment period. Ninety patients were randomized, 45 to each arm. Of the fostamatinib‐treated patients, 35.6% achieved a durable Hgb response versus 26.7% on placebo (p = .398). A post hoc analysis revealed a large placebo response in Eastern European patients. Significantly more patients on fostamatinib from North America, Australia and Western Europe exhibited a durable Hgb response compared to placebo (36% vs. 10.7%, p = .030). After censoring for Hgb values impacted by steroid rescue received during screening and excluding 2 placebo patients found to likely not have wAIHA, a reanalysis demonstrated a difference in durable Hgb response between fostamatinib and placebo (15/45 [33.3%] vs. 6/43 [14.0%], p = .0395). At least 1 AE was reported in 42 (93.3%) and 40 (88.9%) patients receiving fostamatinib and placebo, respectively. The most common AEs in the fostamatinib group were diarrhea (26.7%), hypertension (24.4%), and fatigue (15.6%). In this study, fostamatinib demonstrated a clinically meaningful benefit for patients in Western regions, and no new safety signals were identified.

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Section: Discussionmentioning

confidence: 99%