Algasem Reem, Zainy Nedaa, Alsabban Essam, Almojalli Hamad, Alhasan Khalid, Ali Tariq, Broering Deiter, Aleid Hassan
Department of Pharmacy, King Faisal Specialist Hospital and Research Centre, Riyadh P.O. Box 3354, Saudi Arabia.
Department of Pediatric Nephrology, King Faisal Specialist Hospital and Research Centre, Riyadh P.O. Box 3354, Saudi Arabia.
Pharmacy (Basel). 2024 Aug 8;12(4):123. doi: 10.3390/pharmacy12040123.
Cystinosis is a rare autosomal recessive disorder in which cystine crystals accumulate within the cellular lysosomes, causing damage to multiple organs. Due to challenges with the stringent cysteamine treatment regimen and side effects, adherence is often sub-optimal. This study aimed to assess the level of adherence to cysteamine therapy among cystinosis patients in Saudi Arabia and its impact on their quality of life. Electronic medical record data of 39 cystinosis patients from the Department of Nephrology at King Faisal Specialist Hospital and Research Center in Saudi Arabia were reviewed, and 25 patients were included in this study. Out of the 25 patients included in the final analysis, 64% ( = 16) were female. The mean age was 19.04 years. Almost all patients (23/25, 92%) were on oral IR cysteamine therapy, and 52% (13/25) were on topical cysteamine eye drop treatment. Of the 15 patients who responded to the Morisky Medication Adherence Scale-8 (MMAS-8) questionnaire, only 4 (26.7%) were highly adherent to cysteamine therapy. Most of the respondents (7/15, 46.7%) showed a medium level of treatment adherence. Based on the medication possession ratio for oral cysteamine, only 6 out of 23 patients (26.1%) were found to be 96-100% adherent. For the cysteamine eye drops, only 5/13 patients (38.4%) were 76-95% adherent. The 36-Item Short Form Health Survey (SF-36) used to assess patients' health-related outcomes showed that their quality of life was affected in the domains of 'social functioning' and 'energy/fatigue.' Despite a small sample size, this study shows sub-optimal adherence to cysteamine treatment in patients from Saudi Arabia. The possible reasons for low treatment adherence could be a high frequency of administration and treatment-related side effects.
胱氨酸病是一种罕见的常染色体隐性疾病，其中胱氨酸晶体在细胞溶酶体内蓄积，对多个器官造成损害。由于严格的半胱胺治疗方案存在挑战以及副作用，患者的依从性往往不理想。本研究旨在评估沙特阿拉伯胱氨酸病患者对半胱胺治疗的依从程度及其对生活质量的影响。回顾了沙特阿拉伯法赫德国王专科医院和研究中心肾脏病科39例胱氨酸病患者的电子病历数据，本研究纳入了25例患者。在最终分析纳入的25例患者中，64%（n = 16）为女性。平均年龄为19.04岁。几乎所有患者（23/25，92%）接受口服即释型半胱胺治疗，52%（13/25）接受局部用半胱胺滴眼液治疗。在对Morisky药物依从性量表-8（MMAS-8）问卷作出回应的15例患者中，只有4例（26.7%）对半胱胺治疗高度依从。大多数受访者（7/15，46.7%）显示出中等程度的治疗依从性。根据口服半胱胺的药物持有率，23例患者中只有6例（26.1%）的依从率为96%-100%。对于半胱胺滴眼液，只有5/13例患者（38.4%）的依从率为76%-95%。用于评估患者健康相关结局的36项简明健康调查（SF-36）显示，他们的生活质量在“社会功能”和“精力/疲劳”领域受到影响。尽管样本量较小，但本研究显示沙特阿拉伯患者对半胱胺治疗的依从性不理想。治疗依从性低的可能原因可能是给药频率高和治疗相关的副作用。
