PDF(Pubmed)
Abstract：
BACKGROUND: The objective of this report is to identify and characterize cases of fibrosing colonopathy, a rare and underrecognized adverse event, associated with cysteamine delayed-release (DR) in patients with nephropathic cystinosis.
METHODS: We searched the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) and the medical literature for postmarketing reports of fibrosing colonopathy associated with cysteamine through August 2, 2023.
RESULTS: We identified four cases of fibrosing colonopathy reported with the use of cysteamine DR. The time to onset ranged from 12 to 31 months. In one case, the patient required surgery to have a resection of a section of the strictured colon and a diverting ileostomy. Fibrosing colonopathy was diagnosed by histopathology in two of the cases.
CONCLUSIONS: Our case series identified the risk of fibrosing colonopathy in patients taking cysteamine DR and prompted regulatory action by the FDA. As outlined in changes to the U.S. prescribing information for cysteamine DR, healthcare professionals should be aware of the potential risk of fibrosing colonopathy with cysteamine DR, especially as symptoms can be non-specific leading to misdiagnosis or delayed diagnosis. If the diagnosis of fibrosing colonopathy is confirmed, consideration should be given to permanently discontinuing cysteamine DR and switching to cysteamine immediate-release treatment.
METHODS: We searched the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) and the medical literature for postmarketing reports of fibrosing colonopathy associated with cysteamine through August 2, 2023.
RESULTS: We identified four cases of fibrosing colonopathy reported with the use of cysteamine DR. The time to onset ranged from 12 to 31 months. In one case, the patient required surgery to have a resection of a section of the strictured colon and a diverting ileostomy. Fibrosing colonopathy was diagnosed by histopathology in two of the cases.
CONCLUSIONS: Our case series identified the risk of fibrosing colonopathy in patients taking cysteamine DR and prompted regulatory action by the FDA. As outlined in changes to the U.S. prescribing information for cysteamine DR, healthcare professionals should be aware of the potential risk of fibrosing colonopathy with cysteamine DR, especially as symptoms can be non-specific leading to misdiagnosis or delayed diagnosis. If the diagnosis of fibrosing colonopathy is confirmed, consideration should be given to permanently discontinuing cysteamine DR and switching to cysteamine immediate-release treatment.
摘要：
背景：本报告的目的是确定和表征纤维化结肠病的病例，罕见且未被充分认识的不良事件，与半胱胺延迟释放（DR）相关的肾病性膀胱炎患者。
方法：我们检索了美国食品和药物管理局不良事件报告系统（FAERS）和医学文献，以获取截至2023年8月2日与半胱胺相关的纤维化结肠病的上市后报告。
结果：我们确定了4例使用半胱胺DR报告的纤维化结肠病。发病时间为12至31个月。在一个案例中,患者需要手术切除一段狭窄的结肠和分流回肠造口术。在其中两个病例中，通过组织病理学诊断出了纤维性结肠病。
结论：我们的病例系列确定了服用半胱胺DR的患者发生纤维化结肠病的风险，并促使FDA采取监管措施。正如美国对半胱胺DR处方信息的更改所概述的那样，医疗保健专业人员应该意识到半胱胺DR纤维化结肠病的潜在风险，特别是由于症状可能是非特异性的，导致误诊或延迟诊断。如果诊断为纤维化结肠病，应考虑永久停用半胱胺DR,改用半胱胺速释治疗.
方法：我们检索了美国食品和药物管理局不良事件报告系统（FAERS）和医学文献，以获取截至2023年8月2日与半胱胺相关的纤维化结肠病的上市后报告。
结果：我们确定了4例使用半胱胺DR报告的纤维化结肠病。发病时间为12至31个月。在一个案例中,患者需要手术切除一段狭窄的结肠和分流回肠造口术。在其中两个病例中，通过组织病理学诊断出了纤维性结肠病。
结论：我们的病例系列确定了服用半胱胺DR的患者发生纤维化结肠病的风险，并促使FDA采取监管措施。正如美国对半胱胺DR处方信息的更改所概述的那样，医疗保健专业人员应该意识到半胱胺DR纤维化结肠病的潜在风险，特别是由于症状可能是非特异性的，导致误诊或延迟诊断。如果诊断为纤维化结肠病，应考虑永久停用半胱胺DR,改用半胱胺速释治疗.
