Acquired Hemophilia: Characteristics and Treatment Outcome of a Standardized Therapeutic Approach in a Singapore Series.

Introduction: Acquired hemophilia is a rare bleeding disorder with significant mortality risk but is yet highly treatable. Patients diagnosed with this condition in our institution have been treated with a standardized approach aimed at detecting and controlling bleeding early and inhibitor elimination.

Objective: Review the effectiveness and treatment outcome of our therapeutic approach to acquired hemophilia patients.

Methods: Consecutive patients diagnosed with acquired hemophilia over a period of 5 years from July 1998 till July 2004 were retrospectively reviewed for their underlying characteristics, responses to treatment, complications and subsequent outcome.

Results: 13 patients were recorded with age ranging from 17 to 89 years (median 66 years) and a male to female ratio of 10:3. Follow-up periods range from 5 to 56 months. All patients presented with variable degree of bleeding. Four patients were given recombinant factor VIIa (from 1 to 5 doses) for the control of life threatening bleeding. Three patients received prothrombin complexes as bypassing agents. One other patient received both agents..All patients received both cyclophosphamide (50–100mg/day) and prednisolone (1 mg/kg/day) for elimination of inhibitors at diagnosis. Six patients received intravenous immunoglobulin of 1 gm/kg/day for 2 days with this decision based on the severity of bleeding. Two early deaths were recorded from retroperitoneal bleeding and cerebral infarct following recombinant factor VIIa therapy, respectively. Another death occurred following sepsis with multiple co-morbidities, while being given immunosuppressive therapy. He was the only patient experiencing significant leucopenia while on cyclophosphamide therapy. Two patients were lost to follow-up subsequently. Of 8 patients evaluable for response to inhibitor eliminating therapy with cyclophosphamide and prednisolone, all but one achieved a complete remission within 7 to 71 days with a median of 41 days. Of these 7 patients, there was one early relapse, which occurred from non-compliance to treatment while it was being tailed down. Remission was successfully reinduced on reintroduction of both cyclophophamide and prednisolone. All other patients have remained in remission after discontinuation of therapy. The patient with failure of complete response had persistent low inhibitor levels without bleeding complications and died after 56 months of follow up from unrelated causes. Overall mortality of evaluable patients was 27% (3 out of 11).

Conclusion: Mortality remains significant in this condition with control of early bleeding risks and treatment complications paramount to survival. For the great majority of patients, our strategy affords effective inhibitor elimination and sustained remission of this condition