Key Points• This study is the first to assess prognostic factors in patients with AHA treated according to a uniform immunosuppressive regimen.• Residual factor VIII activity and inhibitor concentration at baseline are potentially useful predictors of remission.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. (Blood. 2015;125(7):1091-1097

Key Points This study is the first to assess the prognostic value of FVIII-specific antibody data in patients with AHA. Anti-FVIII IgA, but not immunoglobulin G, autoantibodies at baseline are potential predictors of recurrence and poor outcome of AHA.

In haemophilic joints with high-grade arthropathy, bleeds occur that do not respond to replacement therapy of the deficient coagulation factor. The reason may be pathologically reactive angiogenesis in chronic synovitis. Seven patients with severe haemophilia A or haemophilia B experienced recurrent massive bleeds of one elbow joint or knee joint in the absence of trauma. After initial application of factor VIII or IX (fVIII/fIX; 50 IU kg(-1) bodyweight), there was only slow and never complete relief of symptoms. Despite intensive secondary prophylaxis maintaining the plasma level of factor concentrate at minimum 50%, new massive bleeds at the same location occurred. Vascular bleeding was suspected. Angiography of the arteries was performed via the femoral artery. Vessels identified as potential bleeding sources were embolized with embolization fluid (ONYX) in eight joints (six elbow and two knee joints). Under low-dose prophylactic treatment (15 IU fVIII or fIX per kg bodyweight for three times per week), no recurrent severe bleed unresponsive to coagulation factor replacement occurred after a mean observation time of 16 months after embolization. The consumption of factor concentrate decreased to one-third of the amount consumed before embolization. In conclusion, angiographic embolization with a non-adhesive liquid embolic agent might be considered as a promising therapeutic and coagulation factor saving option in joint bleeds not responding to replacement of coagulation factor to normal levels.

ZusammenfassungAngeborene Störungen der Thrombozytenfunktion sind eine heterogene Gruppe von Erkrankungen, die oft erst bei Auftreten von Blutungen erkannt werden. Im klinischen Bereich haben sich nur wenige Methoden zur Diagnose und Klassifizierung von angeborenen Thrombozytenfunktionsstörungen bewährt. Für eine rationelle Diagnostik ist ein stufenweises Vorgehen empfehlenswert. Anamnese und klinische Untersuchung sind Grundvoraussetzungen. Das von-Willebrand-Syndrom und andere plasmatische Gerinnungsstörungen sollten vor einer spezifischen Thrombozytenfunktionsdiagnostik immer ausgeschlossen werden. Die Bestimmung von Zahl, Größe, Volumen (MPV) und Morphologie der Thrombozyten erlauben Rückschlüsse auf die zu Grunde liegende Störung.Die PFA-100®-Verschlusszeit eignet sich als Screening zum Ausschluss schwerer Thrombozytenfunktionsstörungen. Die Aggrego metrie ermöglicht die Untersuchung zahlreicher Aspekte der Thrombozytenfunktion. Die Durchflusszytometrie ist zur Diagnose von Thrombasthenie Glanzmann, Bernard-Soulier- Syndrom und Freisetzungsstörungen geeignet. Molekulargenetische Untersuchungen können die Verdachtsdiagnose bestätigen oder zum Nachweis nicht beschriebener Defekte verwendet werden. Hier wird die ungekürzte Version der inter -disziplinären Leitlinie* präsentiert.

Beriate(®) P has an excellent efficacy and safety profile. Many patients who were initiated on Beriate(®) P at our centre remain on the treatment today.