Outcome of CARE: a 6-year national registry of acquired haemophilia A in China

Br J Haematol. 2019 Dec;187(5):653-665. doi: 10.1111/bjh.16128. Epub 2019 Aug 2.

Abstract

Acquired haemophilia A (AHA) is a rare haemorrhagic disorder caused by autoantibodies directed against the functional epitopes of coagulation factor VIII (FVIII). Its management relies on prompt diagnosis, control of bleeding and eradication of the inhibitor by immunosuppression. China Acquired Hemophilia Registry (CARE), a nationwide multicentre registry, was intended to survey the status of AHA and standardize its diagnosis and therapy in China. One hundred and eighty-seven registered patients had an average age of 52 years. Diagnosis was delayed in 46·5% patients. There was a significant delay from diagnosis to immunosuppressive therapy in 68·3% patients. Bleeding control was significantly higher in patients treated with prothrombin complex concentrate (PCC) versus FVIII replacement therapy (84·6% vs. 34·4%; P < 0·001). Inhibitor eradication with a combination of steroids and cyclophosphamide showed a higher partial remission (PR) rate (92·2% vs. 70·3%) and stable complete remission (CR) rate (82·8% vs. 48·6%) than with steroids alone. Logistic regression model showed age and malignancy were significantly related to survival at final follow-up. The mean age for the survivors [51 years (IQR, 35-65 years)] was significantly lower than that of the non-survivors [79 years (IQR, 67-86 years)] (P < 0·001). Overall survival was higher in non-malignancy group than malignancy group (94·9% vs. 70%) (OR = 1·313; 95% CI, 0·913-1·889, P = 0·015).

Keywords: acquired haemophilia A; diagnosis; haemostatic treatment; immunosuppression; outcome; registry.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Age Distribution
  • Aged
  • Aged, 80 and over
  • Autoantibodies / blood
  • Autoimmune Diseases / complications
  • Autoimmune Diseases / epidemiology
  • Blood Coagulation Factors / adverse effects
  • Blood Coagulation Factors / therapeutic use
  • China / epidemiology
  • Databases, Factual
  • Drug Therapy, Combination
  • Factor VIII / immunology
  • Female
  • Glucocorticoids / therapeutic use
  • Hemophilia A / diagnosis*
  • Hemophilia A / drug therapy*
  • Hemophilia A / epidemiology
  • Hemophilia A / etiology
  • Hemorrhage / drug therapy
  • Hemorrhage / epidemiology
  • Hemorrhage / etiology
  • Hemostatics / therapeutic use
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Middle Aged
  • Neoplasms / complications
  • Neoplasms / epidemiology
  • Recurrence
  • Registries
  • Remission Induction
  • Treatment Outcome
  • Young Adult

Substances

  • Autoantibodies
  • Blood Coagulation Factors
  • Glucocorticoids
  • Hemostatics
  • Immunosuppressive Agents
  • prothrombin complex concentrates
  • Factor VIII