ACQUIRED HEMOPHILIA: ANALYSIS OF THE HEMOPHILIA AND THROMBOSIS RESEARCH SOCIETY (HTRS) REGISTRY
Abstract number: O-T-021
Sood1 S.L., Shihong1 I., Swavola1 J., Thielke2 R., Armstrong3 A.E., Gill2 J.C., Kessler4 C.M., Konkle1 B.A., HTRS Registry Investigators5
11Penn Comprehensive Hemophilia and Thrombosis Center, University of Pennsylvania, Philadelphia 22Coagulation Disorders, The Blood Center and Medical College of Wisconsin, Milwaukee, United States 33Coagulation Disorders, Helsinki University Central Hospital, Helsinki, Finland 44Hemophilia and Thrombosis Center, Georgetown University Medical Center, Washington DC 55Hemophilia Treatment Center, Multiple Institutions, Multiple Cities, United States
How-to-cite Sood SL, Shihong I, Swavola J, Thielke R, Armstrong AE, Gill JC, Kessler CM, Konkle BA, HTRS Registry Investigators. ACQUIRED HEMOPHILIA: ANALYSIS OF THE HEMOPHILIA AND THROMBOSIS RESEARCH SOCIETY (HTRS) REGISTRY. J Thromb Haemost 2007; 5 Supplement 2: O-T-021
Introduction: Acquired hemophilia, caused by auto-antibodies to FVIII, is a rare but life-threatening bleeding diathesis. The HTRS database documents treatment patterns in North America.
Methods: 38 patients from the HTRS Database and 21 treated at our HTC were identified (59 total).
Results: Median age at diagnosis was 70 (13-95). Underlying etiologies include: autoimmune (17%), malignant (8%), postpartum (5%), and idiopathic (70%). 96 bleeds occurred: 53 spontaneous (55%), 19 traumatic (20%), 13 postoperative (13%), 10 at IV sites (11%). The most common bleeding site was soft tissue (54%). Median FVIII level on presentation was 1 (0-96) and inhibitor titer 17 BU (0.5-1126). FVIII concentrates with VWF (12%) or without VWF (5%), porcine FVIII (13%), and bypassing agents rFVIIa (52%) or aPCCs (18%) were used as initial treatment for bleeding. No significant difference was found between groups in stopping a bleed within 72h, although trend favored rFVIIa. rFVIIa (52%), aPCCs (47%) and porcine FVIII (48%) appear equally effective in high titer inhibitors. 29 patients achieved CR (52%), 13 (23%) PR, and 14 (25%) no response. Median time to CR was 105 d (9-416). A third of patients with no response died within 3 mo of diagnosis. Overall mortality rate was 19% (11/59) with median follow-up time 11 mo (0-560). All patients with CR were treated with at least 1 form of IS; initial choice was steroid alone (30%), or steroid with cyclophosphamide (40%), rituximab (5%), IVIG (3%), or other combination (22%). No significant difference in CR rate was seen between groups. Age and etiology were significant predictors of CR. Multivariate regression found age, achievement of CR, and bleeding status at 72h as significant predictors of overall survival.
Conclusions: With treatment, the majority of patients with acquired hemophilia are able to achieve a CR. A trend towards more effective control of bleeds with rFVIIa was seen. Each initial immunosuppressive regimen appeared equivalent for achievement of CR in this patient sample.