Patel Nehal, Pandav Ghosha, Thanvi Rashmi, Solanki Priyanka, Shah Dipti, Katara Anil
Department of Pediatrics, Gujarat Medical Education and Research Society (GMERS) Medical College Hospital, Sola, Ahmedabad, IND.
Cureus. 2025 Apr 28;17(4):e83158. doi: 10.7759/cureus.83158. eCollection 2025 Apr.
Regular prophylactic factor replacement is recommended as optimal therapy for hemophilia B. Factor concentrate with longer half-lives would allow successful prophylaxis as less frequent dosing would be less burdensome for patients and caregivers. Nonacog Beta Pegol (N9-GP), a glycopegylated factor IX with an extended half-life, has been established globally. However, real-world data from India remain limited. This study evaluates the safety, efficacy, and feasibility of low-dose N9-GP prophylaxis in Indian children/adolescents with hemophilia B.
In this retrospective study, a total of seven patients with hemophilia B were included. Baseline assessments of the annualized bleeding rate (ABR), Functional Independence Score in Hemophilia (FISH), and Hemophilia Joint Health Score (HJHS) were conducted for all patients. Dosing was individualized (mean 34.76 IU/kg; range: 25-40 IU/kg every three weeks) based on patient weight, baseline ABR, and joint status, in accordance with resource-conscious protocols suitable for Indian settings. Patients were monitored for any adverse events throughout the study period. After 12 months of treatment, the ABR, FISH, and HJHS were reassessed and compared with baseline values. Screening for inhibitors was performed for all patients at the end of the 12-month period.
The mean age was 11.2 years, ranging from four years to 18 years. The mean overall ABR before using N9-GP was 21.1 (range 12 - 48) bleeds/year. After treatment with N9-GP, the mean overall ABR was 0.42 (0-1) bleeds/year. Six patients had target joints before starting treatment. At the end of 12 months of prophylaxis, only one patient had a target joint involving one joint with mobility and swelling significantly improved in the affected joint. Four patients remained bleed free on N9-GP prophylaxis. Three patients experienced minor bleeding once, which was just before the next dose of prophylaxis. Baseline mean HJHS was 26.71/124 (range 10 - 47). After 12 months on N9-GP, mean HJHS was 7.5/124 (range 0-16). Before treatment, the mean FISH score was 23.8/32 (range 17-29). After treatment, the mean FISH score was 31.4/32 (range 28 - 31). None of them developed any adverse drug reactions or thromboembolic events during the entire course of treatment. None of the patients developed inhibitors. Conclusion: N9-GP has been shown to be well tolerated, safe, and efficacious in the treatment and prevention of bleeding episodes in people with hemophilia B.
推荐定期预防性因子替代治疗作为B型血友病的最佳治疗方法。半衰期更长的因子浓缩物可实现成功的预防，因为给药频率降低对患者和护理人员的负担会更小。聚乙二醇化因子IX非阿考糖β（N9-GP）半衰期延长，已在全球范围内获批使用。然而，来自印度的真实世界数据仍然有限。本研究评估低剂量N9-GP预防治疗印度B型血友病儿童/青少年的安全性、有效性和可行性。
在这项回顾性研究中，共纳入了7例B型血友病患者。对所有患者进行了年化出血率（ABR）、血友病功能独立性评分（FISH）和血友病关节健康评分（HJHS）的基线评估。根据患者体重、基线ABR和关节状况，按照适合印度国情的资源节约方案进行个体化给药（平均34.76 IU/kg；范围：每三周25 - 40 IU/kg）。在整个研究期间对患者进行任何不良事件的监测。治疗12个月后，重新评估ABR、FISH和HJHS，并与基线值进行比较。在12个月疗程结束时对所有患者进行抑制剂筛查。
平均年龄为11.2岁，范围为4岁至18岁。使用N9-GP前的平均总体ABR为每年21.1次（范围12 - 48次）出血。用N9-GP治疗后，平均总体ABR为每年0.42次（0 - 1次）出血。6例患者在开始治疗前有目标关节。在预防治疗12个月结束时，只有1例患者有一个目标关节，受累关节的活动度和肿胀明显改善。4例患者在N9-GP预防治疗期间无出血。3例患者有一次轻微出血，均在下一次预防给药前。基线平均HJHS为26.71/124（范围10 - 47）。使用N9-GP 12个月后，平均HJHS为7.5/124（范围0 - 16）。治疗前，平均FISH评分为23.8/32（范围17 - 29）。治疗后，平均FISH评分为31.4/32（范围28 - 31）。在整个治疗过程中，他们均未发生任何药物不良反应或血栓栓塞事件。所有患者均未产生抑制剂。
N9-GP已被证明在治疗和预防B型血友病患者出血发作方面耐受性良好、安全且有效。
