Acquired hemophilia: a single-center survey with emphasis on immunotherapy and treatment-related side-effects.
OBJECTIVE
Acquired hemophilia is a rare disease caused by the development of autoantibodies against factor VIII. Since 1981 we have observed 17 patients with this disorder in our institution. The objective of this survey was to assess the epidemiological features, clinical course, and mortality rate of these patients, with special emphasis on therapy-related side-effects. Also, we present our results with an immunosuppressive approach based on the severity of bleeding episodes.


METHODS
Clinical records of all patients with acquired hemophilia due to factor VIII inhibitor admitted or referred to our hospital between 1981 and 2001 were reviewed retrospectively. We collected each patient's sex, age, medical history, presenting symptoms, activated partial thromboplastin time, factor VIII activity, and inhibitor titre. Patient's clinical courses, including their bleeding episodes, response to therapy, and therapy-related side-effects, were also recorded.


RESULTS
Complete and partial responses were achieved in 14 and one patient, respectively (overall response rate 88%) after a median time to complete response of 3.5 months (range 30 d - 25 months). The inhibitor-related and overall mortality rates were 12% and 29%, respectively. Side-effects were frequent: two patients had blood-borne infections, three patients had thrombotic complications, and nine patients had immunosuppressive therapy-related side-effects. In five patients, discontinuation of cyclophosphamide or prednisone was required.


CONCLUSIONS
Although our response rates were remarkable, this survey showed that treatment-related morbidity could also be very important. Therefore, it is pertinent to bear in mind these potential side-effects in order to decide the most appropriate therapy for each particular patient.
Delgado
Julio
J
Department of Hematology, Hospital Universitario La Paz, Madrid, Spain. jdelgon@terra.ed
Villar
Ana
A
Jimenez-Yuste
Victor
V
Gago
Jose
J
Quintana
Manuel
M
Hernandez-Navarro
Fernando
F
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001323:Autoantibodies / Q000276:immunology; D001327:Autoimmune Diseases / Q000276:immunology / Q000628:therapy; D002648:Child; D005169:Factor VIII / Q000276:immunology*; D005260:Female; D006467:Hemophilia A / Q000209:etiology / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007167:Immunotherapy; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D016019:Survival Analysis
D001323:Autoantibodies; D007166:Immunosuppressive Agents; D005169:Factor VIII