重型血友病A患儿规律低剂量预防治疗后远期健康相关生活质量及相关因素分析

Long-term health-related quality of life and related factors in children with severe hemophilia A who received regular low-dose prophylaxiss

目的探究重型血友病A(HA)患儿规律低剂量预防治疗后远期健康相关生活质量(HRQoL)及相关因素。方法回顾性收集2008年1月1日至2011年12月31日就诊于北京协和医院、开始并规律接受低剂量凝血因子Ⅷ(FⅧ)预防治疗的重型HA患儿的临床资料。最长末次随访时间为2023年5月31日,调查其末次随访时上学或工作的出勤情况、日常运动情况,根据出勤情况分为全勤组、非全勤组;根据运动情况分为运动达标组(达到我国全民体育运动推荐量)、运动不达标组。采用Barthel评分评估日常生活活动能力,采用血友病生活质量问卷(Haemo-QoL)评估生活质量,8~16岁儿童、17岁及以上患者分别使用Haemo-QoL SF、Haem-A-QoL版本,全面评价其远期HRQoL。采用Spearman相关性分析检验治疗情况与Haemo-QoL分值的相关性。结果共纳入22例患儿,开始规律接受低剂量预防治疗的年龄为1.8~17.9(10.4±3.8)岁,低剂量预防治疗期间FⅧ每周平均预防剂量为24.2 U/kg,随访6.3~15.1(9.6±2.8)年。末次随访时,患儿年龄为(20.2±5.4)岁,其中已满18岁成人患者14例(63.6%)。全勤组共15例,非全勤组共7例患者。与全勤组相比,非全勤组预防治疗剂量较小[M(Q 1,Q 3),(28.4±11.1)比(15.3±3.7)U/kg,P=0.012]、预防治疗时间较短[148.1(18.6,346.5)比48.0(32.0,156.9)周,P=0.017]、年度关节出血次数(AJBR)较多[12.5(6.0,22.3)比14.2(13.2,17.8)次,P=0.017]。运动达标组有7例,运动不达标组有15例。与运动达标组比,运动不达标组的预防治疗时间较短[313.7(156.9,366.0)比48.0(16.5,108.9)周,P=0.006]、AJBR较多[7.0(5.1,10.0)比23.3(12.5,29.8),P=0.003]、血友病健康关节评分较高[9.0(2.0,15.5)比23.0(12.0,27.8)分,P=0.014]。Barthel评分显示18例(81.8%)患者日常生活能力不受影响。Haemo-QoL评分中,7例Haemo-QoL SF总分(47.6±17.0)分,15例Haem-A-QoL总分(45.2±22.6)分;Haem-A-QoL评分中日常活动分量表得分最低[38.2(10.0,45.5)分],与预防治疗开始年龄呈正相关(r=0.501,P=0.057),与预防治疗时间呈负相关(r=-0.545,P=0.036)。结论规律低剂量预防治疗可以改善部分重型HA患儿的远期HRQoL,预防剂量高、预防治疗时间长的患儿的生活质量更高。

Objective To investigate long-term health-related quality of life(HRQoL)and related factors in children with severe hemophilia A(HA)who received regular low-dose prophylaxis.Methods Clinical data of severe HA children who began to receive regular low-dose coagulation factorⅧ(FⅧ)prophylaxis in Peking Union Medical College Hospital from January 1,2008 to December 31,2011 were retrospectively enrolled.The longest last follow-up period was May 31,2023.The attendance of school or work and daily physical activity during the last follow-up were investigated.The patients were divided into full attendance group and incomplete attendence group according to attendance.The patients were divided into into exercise attainment group(reached Chinese sports recommendation)and exercise nonattainment group according to the exercise status.Barthel score was used to assess activities of daily living and Haemo-QoL was used to assess quality of life.Long-term HRQoL for children aged 8-16 years and patients aged 17 years and above were assessed using Haemo-QoL SF and Haem-A-QoL versions,respectively.Spearman correlation analysis was used to examine the correlation between treatment conditions and Haemo-QoL scores.Results A total of 22 cases were enrolled,the prophylaxis initiation age ranged from 1.8-17.9(10.4±3.8)years old.The average prophylactic FⅧdose during low-dose prophylaxis was 24.2 U/kg per week and the follow-up time was 6.3-15.1(9.6±2.8)years.At the last follow-up,the age of the patients was(20.2±5.4)years,of which 14(63.6%)were adults over 18 years old.There were 15 patients in the full attendance group and 7 patients in the incomplete attendence group.Compared with the full attendance group,the incomplete attendence group had a smaller preventive treatment dose[M(Q 1,Q 3),(28.4±11.1)vs(15.3±3.7)U/kg,P=0.012],shorter preventive treatment time[148.1(18.6,346.5)vs 48.0(32.0,156.9)weeks,P=0.017],and higher annual joint bleeding rate(AJBR)[12.5(6.0,22.3)vs 14.2(13.2,17.8)times,P=0.017].There were 7 cases in the exercise attainment group and 15 cases in the exercise nonattainment group.Compared to the exercise attainment group,the exercise nonattainment group had shorter preventive treatment time[313.7(156.9,366.0)vs 48.0(16.5,108.9)weeks,P=0.006],a higher AJBR[7.0(5.1,10.0)vs 23.3(12.5,29.8),P=0.003]and a higher hemophilia joint health score(HJHS)[9.0(2.0,15.5)vs 23.0(12.0,27.8),P=0.014].Barthel score showed 81.8%(18 cases)of the patients′living ability was not influenced by the illness.In Haemo-QoL score,the total score of Haemo-QoL SF in 7 cases was(47.6±17.0)scores,the total score of Haem-A-QoL in 15 cases was(45.2±22.6)scores.The daily activity dimension of the Haem-A-QoL score was the lowest[38.2(10.9,45.5)scores],which was positively correlated with the starting age of prophylactic initiation(r=0.501,P=0.057),and negatively correlated with the duration of prophylaxis(r=-0.545,P=0.036).Conclusions Regular low-dose prophylaxis could improve the long-term HRQoL of some children with severe HA,and children with higher prophylactic doses and longer prophylactic treatment time have higher quality of life.