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Autoimmune hemolytic anemia (AIHA)

Autoimmune hemolytic anemia (AIHA) occurs when antibodies directed against the person's own red blood cells (RBCs) cause them to lyse, leading to an insufficient number of oxygen-carrying red blood cells in the circulation

Prevalence

17 / 100,000

3,310 - 29,790

US Estimated

5,135 - 46,215

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 know 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: AcalabrutinibClick here for more information
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
Click here for more information
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: rilzabrutinibClick here for more information
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
Click here for more information
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: IsatuximabClick here for more information
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: ParsaclisibClick here for more information
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-2Click here for more information
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: FostamatinibClick here for more information
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: BIVB020Click here for more information

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).AstraZenecaClick here for more informationClick here for more information
ParsaclisibParsaclisib is an inhibitor of the delta isoform of phosphoinositide-3 kinase (PI3K) with potential antineoplastic activityPhase 2IncyteClick here for more informationClick here for more information
IsatuximabMultimodal Anti-CD38 Monoclonal AntibodyPhase 1/2 -Marketed as Sarclisa for multiple myeloma.Sanofi GenzymeClick here for more informationClick here for more information
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 1/2JanssenClick here for more informationClick here for more information
rilzabrutinibRizabrutinib is a small-molecule inhibitor of Bruton's tyrosine kinase (BTK)Phase 3Sanofi GenzymeClick here for more informationClick here for more information
APL-2Complement (C3) InhibitorPhase 2ApellisClick here for more informationClick here for more information
BIVB020BIVV020 is an investigational monoclonal antibody that targets Complement C1sPhase 1/2Bioverativ, a Sanofi companyClick here for more informationClick here for more information
FostamatinibFostamatinib is a small molecule Syk kinase inhibitor with potential anti-inflammatory and immunomodulating activitiesPhase 3 - Marketed as Tavalisse for ITP.Rigel PharmaceuticalsClick here for more informationClick here for more information

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