Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study

TY - JOUR

T1 - Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study

AU - Tiede, Andreas

AU - Klamroth, Robert

AU - Scharf, Rüdiger E

AU - Trappe, Ralf U

AU - Holstein, Katharina

AU - Huth-Kühne, Angela

AU - Gottstein, Saskia

AU - Geisen, Ulrich

AU - Schenk, Joachim

AU - Scholz, Ute

AU - Schilling, Kristina

AU - Neumeister, Peter

AU - Miesbach, Wolfgang

AU - Manner, Daniela

AU - Greil, Richard

AU - von Auer, Charis

AU - Krause, Manuela

AU - Leimkühler, Klaus

AU - Kalus, Ulrich

AU - Blumtritt, Jan-Malte

AU - Werwitzke, Sonja

AU - Budde, Eva

AU - Koch, Armin

AU - Knöbl, Paul

N1 - © 2015 by The American Society of Hematology.

PY - 2015/2/12

Y1 - 2015/2/12

N2 - Acquired hemophilia A (AHA) is caused by autoantibodies against factor VIII (FVIII). Immunosuppressive treatment (IST) results in remission of disease in 60% to 80% of patients over a period of days to months. IST is associated with frequent adverse events, including infections as a leading cause of death. Predictors of time to remission could help guide IST intensity but have not been established. We analyzed prognostic factors in 102 prospectively enrolled patients treated with a uniform IST protocol. Partial remission (PR; defined as no active bleeding, FVIII restored >50 IU/dL, hemostatic treatment stopped >24 hours) was achieved by 83% of patients after a median of 31 days (range 7-362). Patients with baseline FVIII <1 IU/dL achieved PR less often and later (77%, 43 days) than patients with ≥1 IU/dL (89%, 24 days). After adjustment for other baseline characteristics, low FVIII remained associated with a lower rate of PR (hazard ratio 0.52, 95% confidence interval 0.33-0.81, P < .01). In contrast, PR achieved on steroids alone within ≤21 days was more common in patients with FVIII ≥1 IU/dL and inhibitor concentration <20 BU/mL (odds ratio 11.2, P < .0001). Low FVIII was also associated with a lower rate of complete remission and decreased survival. In conclusion, presenting FVIII and inhibitor concentration are potentially useful to tailor IST in AHA.

AB - Acquired hemophilia A (AHA) is caused by autoantibodies against factor VIII (FVIII). Immunosuppressive treatment (IST) results in remission of disease in 60% to 80% of patients over a period of days to months. IST is associated with frequent adverse events, including infections as a leading cause of death. Predictors of time to remission could help guide IST intensity but have not been established. We analyzed prognostic factors in 102 prospectively enrolled patients treated with a uniform IST protocol. Partial remission (PR; defined as no active bleeding, FVIII restored >50 IU/dL, hemostatic treatment stopped >24 hours) was achieved by 83% of patients after a median of 31 days (range 7-362). Patients with baseline FVIII <1 IU/dL achieved PR less often and later (77%, 43 days) than patients with ≥1 IU/dL (89%, 24 days). After adjustment for other baseline characteristics, low FVIII remained associated with a lower rate of PR (hazard ratio 0.52, 95% confidence interval 0.33-0.81, P < .01). In contrast, PR achieved on steroids alone within ≤21 days was more common in patients with FVIII ≥1 IU/dL and inhibitor concentration <20 BU/mL (odds ratio 11.2, P < .0001). Low FVIII was also associated with a lower rate of complete remission and decreased survival. In conclusion, presenting FVIII and inhibitor concentration are potentially useful to tailor IST in AHA.

U2 - 10.1182/blood-2014-07-587089

DO - 10.1182/blood-2014-07-587089

M3 - SCORING: Journal article

C2 - 25525118

VL - 125

SP - 1091

EP - 1097

JO - BLOOD

JF - BLOOD

SN - 0006-4971

IS - 7

ER -