A recombinant coagulation factor IX albumin fusion protein (rIX-FP, CSL654) was developed with an improved PK profile to support a longer dosing interval for routine prophylaxis treatment. Japanese subjects have participated in a Study CSL654_3001, phase 3 open label, multinational, pivotal study of rIX-FP. Male hemophilia B patients with no inhibitor, 12–65 years of age, at least 150 exposure days with previous FIX products were eligible for enrollment. 10 of the 63 Study 3001 subjects were recruited in Japan. The mean t_1/2 of rIX-FP in Japanese and all subjects were 95 h and 102 h respectively. The mean annualized spontaneous bleeding rate in Japanese and all subjects were 12.8 and 14.6 respectively for on-demand treatment, 1.8 and 0.6 respectively for weekly prophylaxis regimen, 0.4 and 1.1 respectively for every 2 weeks prophylaxis regimen. No patient developed an inhibitor and no safety concerns were identified in Study 3001. The results of Japanese subjects were consistent with those observed for the global population. rIX-FP is safe and effective for preventing bleeding episodes at weekly and every 2 weeks dosing regimens in Japanese patients with hemophilia B.

A questionnaire-based survey to assess clinicians’ attitudes toward acquired hemophilia A (AHA) was conducted to determine if clinicians recognized the condition and to improve the prognosis of AHA in 8 hospitals in Niigata Prefecture, Japan. Degree of recognition of AHA was about 60%, which was higher in foundation hospitals. Main knowledge source of AHA was daily clinic in foundation hospital and books in small and medium sized hospitals. Seventy two percent of doctors who did not know AHA wanted to know AHA. Sixteen percent of all doctors encountered AHA, with the frequency of encounter being the highest for physicians and second highest for orthopedists. Only 5% of the doctors had experience with treating AHA. Approximately 83% of doctors wanted to refer patients with AHA to a hematologist. Sixty percent of doctors who responded wanted to refer patients with AHA to other hospitals or other departments like hemophilia center hospital. It is important to know the consultation department in order to improve a medical cooperation system to the department of hematology and in order to improve prognosis of AHA.