Disease-modifying treatments for primary autoimmune haemolytic anaemia

Liu, Anthony Pak-Yin; Cheuk, Dkl

doi:10.1002/14651858.cd012493

Cited by 3 publications

(2 citation statements)

References 21 publications

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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%

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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“…A anemia hemolítica autoimune (AHAI) é uma doença na qual os pacientes produzem anticorpos anti-eritrócitos contra as glicoproteínas de membrana absorvidas na superfície dos eritrócitos. Sua etiopatologia ainda não foi completamente esclarecida, podendo existir relação com o uso de medicamentos, infecções virais e associação com outras doenças autoimunes 65,66 .…”

Section: Anemia Hemolítica Autoimuneunclassified

Fisiopatogenia e métodos diagnósticos das anemias hemolíticas: uma revisão integrativa.

Cruz¹,

Antunes²

2018

SDH

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Introdução: Hemoglobinopatias decorrem de mutações nos genes responsáveis pela síntese das cadeias globínicas e possuem padrão de herança autossômico recessivo. As alterações estruturais detectadas mundialmente nas cadeias globínicas incluem hemoglobina S, hemoglobina C, hemoglobina E e hemoglobina D. Objetivo: Revisar a literatura sobre as principais hemoglobinopatias abordando a sua fisiopatogenia, métodos diagnósticos e alterações laboratoriais. Métodos: Adotou-se descritores específicos (inglês/português) vinculados ao recurso “MeshDatabase” e ao operador booleano (AND/OR). As buscas foram realizadas no PUBMED, LILACS, ScIELO e COCHRANE. Os descritores “anemias hemolíticas, hemoglobinopatias e diagnóstico laboratorial” deveriam constar no título, e/ou palavras-chave e/ou resumo. Além disso, foram incluídos apenas os estudos entre 2012 e 2017. Resultados: Foram identificadas 1.010 referências, e, destas, 937 foram considerados elegíveis para inclusão. Ao final, 79 foram incluídos no estudo. Conclusões: Considerando-se a importância de um diagnóstico laboratorial preciso e fidedigno com a clínica apresentada pelo paciente, o presente estudo revisou a literatura científica identificando as principais hemoglobinopatias e caracterizando as alterações quantitativas e qualitativas nas principais anemias hemolíticas. É importante realizar e interpretar corretamente esses exames, e, sempre que possível, incluir, no laudo do paciente, observações que podem auxiliar no manejo clínico.

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