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Autoimmune hemolytic anemia (AIHA)

Autoimmune hemolytic anemia (AIHA) is an acquired, heterogeneous group of diseases which includes warm AIHA, cold agglutinin disease (CAD), mixed AIHA, paroxysmal cold hemoglobinuria and atypical AIHA

Prevalence

17 / 100,000

56,270

US Estimated

87,295

Europe Estimated

Age of Onset

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ICD-10

D59.0
D59.1

Inheritance

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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X-linked dominant

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X-linked recessive

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Rare View

Autoimmune hemolytic anemia (AIHA) is an acquired, heterogeneous group of diseases which includes warm AIHA, cold agglutinin disease (CAD), mixed AIHA, paroxysmal cold hemoglobinuria and atypical AIHA.
Autoimmune hemolytic anemia (AIHA) occurs when antibodies directed against red blood cells (RBCs) lyse, leading to an insufficient number of oxygen-carrying red blood cells in the circulation. The lifetime of the RBCs is reduced from the normal 100–120 days to just a few days in serious cases.

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5 Facts you should know

FACT

1

Autoimmune hemolytic anemia (AIHA) occurs when antibodies directed against the person's own red blood cells (RBCs) lyse

FACT

2

The lifetime of the RBCs is reduced from the normal 100–120 days to just a few days in serious cases

FACT

3

Symptoms include unusual weakness and fatigue with tachycardia and breathing difficulties, jaundice, dark urine and/or splenomegaly

FACT

4

There are two main types of autoimmune hemolytic anemia: warm antibody hemolytic anemia and cold antibody hemolytic anemia

FACT

5

Autoimmune hemolysis may be a precursor of later onset systemic lupus erythematosus

Autoimmune hemolytic anemia (AIHA) is also known as...

Autoimmune hemolytic anemia (AIHA)

Anemia hemolytic autoimmune; Acquired autoimmune hemolytic anemia; Idiopathic autoimmune hemolytic anemia, immunohemolytic anemia

What’s your Rare IQ?

Which of the following is not a typical symptom of AIHA?

Common signs & symptoms

Abnormal leukocyte morphology

Autoimmunity

Autoimmune disease

Dyspnea

Trouble breathing

Fatigue

Headache

Hemolytic anemia

Muscle weakness

Pallor

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsMore Information
Acalabrutinib for the Treatment of Relapsed or Refractory Autoimmune Hemolytic Anemia in Patients With Chronic Lymphocytic LeukemiaThis phase II trial studies the effect of acalabrutinib in treating autoimmune hemolytic anemia that has come back (relapsed) or has not responded to previous treatment (refractory) in patients with chronic lymphocytic leukemia. Acalabrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.Phase 2RecruitingDrug: AcalabrutinibMore Info
Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic AnemiaThe main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).Phase 2/3RecruitingDrug: M281 (Nipocalimab)
Drug: Placebo
More Info
Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)This is a single group treatment, Phase 2, open-label, study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in adult patients with wAIHA.Phase 2RecruitingDrug: rilzabrutinibMore Info
Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia (PATHWAY)The purpose of this study is to evaluate the efficacy and safety of parsclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA)Phase 3RecruitingDrug: parsaclisinib
Drug: placebo
More Info
Safety, Pharmacokinetics, and Efficacy of Subcutaneous Isatuximab in Adults With Warm Autoimmune Hemolytic Anemia (wAIHA)Part A: To evaluate the safety and tolerability of subcutaneous injections of isatuximab in adults with wAIHA
Part B: To evaluate the efficacy of the selected dose in adults with wAIHA
Phase 1/2RecruitingDrug: IsatuximabMore Info
A Study of INCB050465 in Participants With Autoimmune Hemolytic AnemiaThe purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that requires treatment intervention.Phase 2Active, not recruitingDrug: ParsaclisibMore Info
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)This study is to assess the safety, tolerability, preliminary efficacy, and pharmacokinetics of APL-2 in subjects with warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD).Phase 2Active, not recruitingDrug: APL-2More Info
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHAThe primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).Phase 3Active, not recruitingDrug: FostamatinibMore Info
A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin DiseaseTo assess the safety and tolerability in participants with cold agglutinin disease (CAD), after a single dose of intravenous (IV) BIVV020Phase 1Active, not recruitingDrug: BIVB020More Info

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
AcalabrutinibAcalabrutinib is a small-molecule inhibitor of Bruton's tyrosine kinase (BTK)Phase 2 - Marketed as Calquence to treat adults with mantle cell lymphoma (MCL) who have received at least one prior treatment for their cancer, or adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).AstraZenecaMore InfoMore Info
ParsaclisibParsaclisib is an inhibitor of the delta isoform of phosphoinositide-3 kinase (PI3K) with potential antineoplastic activityPhase 2IncyteMore InfoMore Info
IsatuximabMultimodal Anti-CD38 Monoclonal AntibodyPhase 1/2 -Marketed as Sarclisa for multiple myeloma.Sanofi GenzymeMore InfoMore Info
NipocalimabNipocalimab has a unique mechanism of action whereby it blocks the IgG binding site on endogenous FcRn and is expected to decrease pathogenic IgG antibody concentrationsPhase 1/2JanssenMore InfoMore Info
rilzabrutinibRizabrutinib is a small-molecule inhibitor of Bruton's tyrosine kinase (BTK)Phase 3Sanofi GenzymeMore InfoMore Info
APL-2Complement (C3) InhibitorPhase 2ApellisMore InfoMore Info
BIVB020BIVV020 is an investigational monoclonal antibody that targets Complement C1sPhase 1/2Bioverativ, a Sanofi companyMore InfoMore Info
FostamatinibFostamatinib is a small molecule Syk kinase inhibitor with potential anti-inflammatory and immunomodulating activitiesPhase 3 - Marketed as Tavalisse for ITP.Rigel PharmaceuticalsMore InfoMore Info

Michalak SS, Olewicz-Gawlik A, Rupa-Matysek J, Wolny-Rokicka E, Nowakowska E, Gil L. Autoimmune hemolytic anemia: current knowledge and perspectives. Immun Ageing. 2020;17(1):38. Published 2020 Nov 20. doi:10.1186/s12979-020-00208-7