title: Treatment and outcome of acquired haemophilia A with a standard conventional regimen in a cohort without associated conditions.
abstract: Acquired haemophilia A (AH) is a rare bleeding disorder with significant risk of mortality. We conducted a study on the treatment and outcomes of patients with AH diagnosed between 1998 and 2004 in our institution. Fourteen patients (age range 17-89, median 64) were followed-up from between 10-64 months (average 32 months). Inhibitor levels ranged from 5 to 450 BU. Ten patients required either recombinant activated factor VIIa or prothrombin complexes for control of bleeding. All patients received a standard immunosuppressive regimen of prednisolone 1 mg kg day(-1) and oral cyclophosphamide 50-100 mg day(-1). In addition, nine patients received intravenous immunoglobulin. Complete remission (CR) was achieved in 88% (eight of nine evaluable patients) over durations ranging from 5-78 days (average 35 days). There were three mortality; from bleeding, sepsis and cerebral infarct. Two patients were lost to follow-up. There were no relapses among patients achieving CR. Conditions associated with AH were not identified at diagnosis or subsequent follow-up in all patients. This series represent one of the largest aetiologically and treatment-wise, uniformed cohort of AH patients. Despite toxicity concerns, the combination of prednisolone and cyclophosphamide remain an effective regimen and should be among the first line of treatment choices, with careful patient selection.
authors.affiliation: Department of Haematology, Singapore General Hospital, Singapore, Singapore. ng.heng.joo@sgh.com.sg
authors.forename: H J
authors.initials: HJ
authors.lastname: Ng
authors.forename: D C L
authors.initials: DC
authors.lastname: Tan
authors.forename: L H
authors.initials: LH
authors.lastname: Lee
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D019774:Blood Coagulation Factor Inhibitors / Q000097:blood; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000009:adverse effects / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005169:Factor VIII / Q000037:antagonists & inhibitors / Q000276:immunology; D015942:Factor VIIa / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006467:Hemophilia A / Q000188:drug therapy* / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use; D007518:Isoantibodies / Q000097:blood; D008297:Male; D008875:Middle Aged; D011239:Prednisolone / Q000627:therapeutic use; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D007166:Immunosuppressive Agents; D007518:Isoantibodies; D011994:Recombinant Proteins; D003520:Cyclophosphamide; D005169:Factor VIII; D011239:Prednisolone; D015942:Factor VIIa