Badawy Sherif M, Thompson Alexis A, Lai Jin-Shei, Penedo Frank J, Rychlik Karen, Liem Robert I
Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
Pediatr Blood Cancer. 2017 Jun;64(6). doi: 10.1002/pbc.26369. Epub 2016 Nov 28.
Complications related to sickle cell disease (SCD) result in significant declines in health-related quality of life (HRQOL). While hydroxyurea reduces SCD complications, adherence remains suboptimal. The study's objectives were to assess the feasibility of Internet-based electronic assessment of HRQOL in SCD clinic and to examine the relationship between HRQOL and hydroxyurea adherence in adolescents and young adults (AYAs) with SCD.
A cross-sectional survey was administered on tablets to 34 AYAs (12-22 years old) in a SCD clinic from January through December 2015. Study measures included Patient Reported Outcomes Measurement Information System (PROMIS ) computerized adaptive testing and ©Modified Morisky Adherence Scale 8-items (©MMAS-8).
Participants (59% male, 91% Black) had median age of 13.5 (range 12-18) years. Ninety-one percent completed PROMIS® measures electronically in the clinic, meeting our feasibility criterion of ≥85% completion rate. ©MMAS-8 scores positively correlated with fetal hemoglobin (HbF) (r = 0.34, P = 0.04) and mean corpuscular volume (MCV) (r = 0.42, P = 0.01) and inversely correlated with fatigue (r = -0.45, P = 0.01), depression (r = -0.3, P = 0.08), and social isolation (r = -0.78, P = 0.02). Low ©MMAS-8 scores, indicating poor adherence, were associated with worse fatigue (P = 0.001) and trended toward significance for pain (P = 0.07) and depression (P = 0.06). Homozygous hemoglobin S disease patients with low HbF (<16%) had worse social isolation (P = 0.04) and those with low MCV (<102 fl) reported worse fatigue (P = 0.001), pain (P = 0.01), mobility (P = 0.01), and social isolation (P = 0.04).
HRQOL assessment in the SCD clinic is feasible. SCD patients with low hydroxyurea adherence and/or low HbF or MCV levels had worse HRQOL scores, particularly fatigue. Future prospective studies examining the relationship between HRQOL and hydroxyurea adherence are warranted.
与镰状细胞病（SCD）相关的并发症导致健康相关生活质量（HRQOL）显著下降。虽然羟基脲可减少SCD并发症，但依从性仍不理想。本研究的目的是评估在SCD诊所基于互联网进行HRQOL电子评估的可行性，并研究青少年和青年（AYA）SCD患者中HRQOL与羟基脲依从性之间的关系。
2015年1月至12月，在一家SCD诊所对34名年龄在12至22岁之间的AYA进行了平板电脑横断面调查。研究指标包括患者报告结局测量信息系统（PROMIS）计算机自适应测试和改良的Morisky依从性量表8项（MMAS-8）。
参与者中男性占59%，黑人占91%，中位年龄为13.5岁（范围12至18岁）。91%的参与者在诊所通过电子方式完成了PROMIS测量，达到了我们≥85%完成率的可行性标准。MMAS-8评分与胎儿血红蛋白（HbF）呈正相关（r = 0.34，P = 0.04）和平均红细胞体积（MCV）呈正相关（r = 0.42，P = 0.01），与疲劳呈负相关（r = -0.45，P = 0.01）、抑郁呈负相关（r = -0.3，P = 0.08）和社交隔离呈负相关（r = -0.78，P = 0.02）。MMAS-8评分低表明依从性差，与更严重的疲劳相关（P = 0.001），疼痛（P = 0.07）和抑郁（P = 0.06）有显著趋势。HbF低（<16%）的纯合血红蛋白S病患者社交隔离更严重（P = 0.04），MCV低（<102 fl）的患者报告有更严重的疲劳（P = 0.001）、疼痛（P = 0.01）、活动能力（P = 0.01）和社交隔离（P = 0.04）。
在SCD诊所进行HRQOL评估是可行的。羟基脲依从性低和/或HbF或MCV水平低的SCD患者HRQOL评分更差，尤其是疲劳。有必要进行未来的前瞻性研究来考察HRQOL与羟基脲依从性之间的关系。
