Sirolimus in the Treatment of Refractory/Relapsed wAIHA
Warm Autoimmune Hemolytic AnemiaAutoimmune hemolytic anemia (AIHA) is a rare and heterogeneous disorder characterized by the destruction of red blood cells through warm or cold antibodies. Glucocorticoid (combined with rituximab) is the first-line treatment. However, the recurrence rate is very high and some patients may not respond to steroids. Second-line therapies include cyclosporine A (CsA), cyclophosphamide, rituximab, azathioprine, and even splenectomy. Our previous study of sirolimus in refractory/relapsed AIHA and ES found an effective rate of 80%. Therefore, the investigators plan to explore the efficacy and safety of sirolimus in the treatment of refractory/relapsed wAIHA.
A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody...
Warm Antibody Autoimmune Hemolytic AnemiaThe primary objective of this study is: • To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
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.
Safety and Tolerability Study in Adults With Cold Agglutinin Disease Previously Treated With SAR445088...
Autoimmune Haemolytic AnaemiaPrimary Objective: To assess the long-term safety and tolerability in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088 Secondary Objective: To assess, in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088: The long-term effect of SAR445088 on complement mediated hemolysis The long-term pharmacodynamics (PD) effect of SAR445088 relating to complement inhibition The long-term pharmacokinetic (PK) profile of SAR445088 The long-term immunogenicity of SAR445088
HMPL-523 (Sovleplenib) in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Warm Antibody Autoimmune Hemolytic AnemiaPhase II Study: To evaluate the safety and preliminary efficacy of HMPL-523 in adult patients with wAIHA Phase III Studies: Confirmation of Efficacy safety and of HMPL-523 in Adult Patients With wAIHA
Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic...
Warm Autoimmune Hemolytic Anemia (wAIHA)The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological...
Immune Thrombocytopenia (ITP)Cold Agglutinin Disease (CAD)The main purpose of this study is to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.
Acalabrutinib for the Treatment of Relapsed or Refractory Autoimmune Hemolytic Anemia in Patients...
Autoimmune Hemolytic AnemiaChronic Lymphocytic Leukemia2 moreThis 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.
Efficacy of Prolonged Anticoagulation for Primary Prevention of Venous Thromboembolic Disease in...
Prolonged AnticoagulationVenous Thromboembolic Disease1 moreAutoimmune hemolytic anemia (AIHA) is a rare autoimmune disease (incidence <1/100,000 population) responsible for the destruction of red blood cells by the host immune system, notably through the action of autoantibodies. Apart from complications related to anemia, the occurrence of venous thromboembolism (VTE) in this population is frequent, estimated at 20-27%. The risk of VTE is highest during the period of hemolysis, especially during the first 3 months after the diagnosis of AIHA. This risk is 7.5 [4.7; 12.0] times greater than in the general population. No clinical predictive factor for VTE was identified and the usual factors (cancer, previous VTE, bed rest >3 days, surgery, age >70 years, heart or respiratory failure, myocardial infarction, stroke, obesity, hormone replacement therapy) were not considered. Several biological risk factors have been suggested (depth of anemia, bilirubin level, leukocyte count, antiphospholipid antibodies) but have not been confirmed in other studies. AIHA is therefore a risk factor for VTE in its own right, and the National Diagnostic and Care Protocol (NDCP) recommends the implementation of VTE prevention during acute hemolysis (Grade C). However, the value of this prophylaxis has never been prospectively evaluated and its duration is empirical. In practice, low-molecular-weight heparin (LMWH) is generally used during "flare-ups" of AIHA (diagnosis and relapse) in hospitalized patients, but is rarely continued beyond the hospital phase when VTE also occurs in ambulatory patients. Thus, we hypothesize that prolonged preventive anticoagulation during the 12-week risk period following diagnosis or relapse of AIHA could decrease the incidence of VTE. In orthopedic surgery, this strategy has been proven to decrease VTE from 50% to 10-15%. In certain high-risk medical situations, prolonged prophylaxis with apixaban has been shown to decrease the occurrence of VTE from 10.2% to 4.2% in solid cancers4 and from 4-11% to 2% in myeloma.
Cold Agglutinin Disease Real World Evidence Registry
Cold Agglutinin Disease (CAD)Cold Agglutinin Syndrome (CAS)This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 [NCT02502903,CAD patients], BIVV009-03/EFC16215 [NCT03347396], and BIVV009-04/EFC16216 [NCT03347422]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.