Severe haemophilia A children on low‐dose tertiary prophylaxis showed less joint deterioration and better maintenance of functional independence than children on on‐demand treatment: A 6‐year follow‐up study

Abstract

Background: In countries with limited resource, haemophilia patients have to choose low-dose prophylaxis or on-demand treatment (ODT) because of economic constraints. Whether low-dose prophylaxis can achieve better joint function outcome than ODT over long-term remains unclear. Aim: To investigate the long-term effect of low-dose tertiary prophylaxis versus ODT on joint health in severe haemophilia A children. Methods: This was a retrospective study. We enrolled and followed 34 severe haemophilia boys in China receiving on-demand treatment (n = 18) or low-dose prophylaxis (10-15 IU/kg, 2-3 times per week) for a medium-term (6-18 months, n = 9) or longer-term (19-30 months, n = 7). We evaluated their haemophilia joint health score (HJHS) 2.1 and functional independence score in haemophilia (FISH) at baseline and at their 6-year follow-up visits. Their annual bleeding rate (ABR) and annual joint bleeding rate (AJBR) were also recorded. Results: During the 6-year follow-up period, ABR and AJBR were similar between the 2 prophylaxis groups, with each of the 2 prophylaxis group rates being significantly better (lower) than the ODT group (P < .05). Compared to baseline values, evaluation at 6-year follow-up showed HJHS improvement in both prophylaxis groups, although significantly (P < .05) only in the longer-term prophylaxis group. The FISH score showed insignificant change in patients in each prophylaxis cohort, compared to significant worsening (P < .05) in the ODT group. Conclusion: Low-dose tertiary prophylaxis reduced ABR and AJBR of children with severe haemophilia and better maintained their functional independence by the FISH over the long term. Longer-term prophylaxis also improved their joint health status by the HJHS.

Low‐dose prophylaxis and its impact on the health of haemophilia patients

“…Twenty‐four patients had zero bleeds at the end of treatment [16]. Liu et al in a 6‐year follow‐up study on medium‐term (6–18 months) versus long‐term prophylaxis (19–30 months) showed significant reduction in both ABR and AJBR in medium‐term as well as long‐term prophylactic groups, but significant differences in HJHS scores were observed only in the long‐term prophylaxis group and not in the medium‐term group [17].…”

“…With limited access to factors for majority of the patients globally, assessment of different dosages/protocols, specifically low-dose prophylaxis (LDP) protocols and pharmacokinetic (PK)-guided dose escalation protocols, are becoming increasingly important. Few reports from developing countries do support the superiority of LDP over ET in terms of annualized bleeding rate (ABR), annual joint bleed rate (AJBR) and the joint scores [7][8][9][10][11][12][13][14][15][16][17][18][19][20][21][22][23][24][25]. However, there is high heterogeneity in these reports in terms of demographics of patients, inclusion criteria, dosages, type of products and the outcome measurement parameters.…”

The effects of close kinetic chain exercises on proprioception and physical activity level in pediatric patients with hemophilia

“…The FISH has also been used to evaluate the effects of pharmacological therapies including prophylaxis on functional independence 35 . In a study assessing functional independence in children with hemophilia A, FISH was used to evaluate musculoskeletal function in 98 patients.…”

Functional evaluation and rehabilitation of musculoskeletal complications in hemophilia

“…Health Score，HJHS) [15] ，适用于血友病早期关节病变评估，在 4-18 岁的儿童及青少年中 进行了信度 [15] 和效度 [16] 此量表虽未在成人血友病患者中进行验证，但也有研究者在成人患者中使用 [19][20][21] 。2019 年， Ribeiro 等人更新了 HJHS 的评分系统，使用加权评分来提高 HJHS 评估的精确度 [22] 。此量表 是目前国内唯一经过信效度验证的关节功能评估量表，量表具体内容，详见电子版附表 1， 因此，国内相关研究均使用此量表 [23][24][25][26][27][28][29][30] 关节出血引起的急性疼痛，减少流向受伤组织的血流量 [51] 。也有研究表明冰敷可能会干扰凝 血，延缓止血过程 [52] 。然而，许多血友病患者喜欢冰敷来缓解疼痛 [53] 。加压包扎可降低再 出血的风险，抬高患肢可减轻肢体的肿胀。但效果通常是短暂的，如果使用这些措施，需要 患者能够耐受并感觉舒适 [54] 。 出血停止后，针对关节积血，还可以进行物理治疗。物理因子如激光 [55] 、脉冲短波 [56] 有助于积血清除，并缓解关节疼痛和肿胀。运动治疗应包括主动和被动的关节活动度练习、 本体感觉训练，直到关节活动和功能完全恢复至出血前状态 [57] 血友病性骨关节病可伴有一定程度的疼痛，应注意和出血引起的疼痛相鉴别，对于骨关 节病引起的疼痛，物理因子具有一定的疗效，如无热量超短波 [64] 、低热量磁疗 [64] 、偏振光 治疗 [65] 、中频治疗 [65] 、冰敷 [65] [8]Rodriguez-MerchanEC . Pathogenesis, early diagnosis, and prophylaxis for chronic hemophilic synovitis .…”