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帕利夫明治疗非造血干细胞移植儿科患者口腔黏膜炎的疗效:一家三级医院的经验

Efficacy of palifermin in the treatment of oral mucositis in non-hematopoietic stem cell transplant pediatric patients: experience of a single tertiary hospital.

作者信息

Hamzi Hasna, Binhassan Amal, Najmeldin Amal, Alhariri Ebtsam, Elhussein Bodour, Althibani Nour, Alenazi Ahmad, Alsharif Shahad, Alshahrani Mohammad, Alsharif Omar, Alkhayat Nawaf, Elborai Yasser

机构信息

Department of Pediatric Hematology/Oncology, Prince Sultan Military Medical City, Riyadh, Saudi Arabia.

Department of Pediatric, Alyamamah Hospital, Riyadh, Saudi Arabia.

出版信息

J Egypt Natl Canc Inst. 2024 Dec 2;36(1):38. doi: 10.1186/s43046-024-00247-x.

Abstract

BACKGROUND

Severe oral mucositis (OM) is one of the adverse events post-chemotherapy, radiation, and stem cell transplantation with major clinical and economic impact. The management of severe OM remains challenging. This study aimed to look for the benefit and clinical impact of palifermin for mucositis among the non-transplanted pediatric cancer population.

METHODS

This is a descriptive retrospective study extended from 2016 to 2020 at Prince Sultan Military Medical City (PSMMC), in Saudi Arabia. During this period all pediatric patients (< 14 years of age) on chemotherapy and complicated by severe OM that required palifermin (35 courses), as off-labeled medication, were analyzed looking for the clinical demographics, primary diagnosis, chemotherapy agents used, effectiveness, and tolerability of palifermin.

RESULTS

A total of 29 patients with severe OM received 35 palifermin courses. All of them received 60 mcg/kg/day IV for 3 consecutive days. 20% of them have acute lymphoblastic leukemia (ALL). We noticed that 60% of severe OM required palifermin post anthracycline while high-dose methotrexate aggravates the risk in 40% of them. Only 25.7% of the patients required TPN for a median duration of 5 days and 54.3% of them received opioids for a median duration of 4 days. Twenty patients (57.1%) had used antibiotics, 4 patients were on antifungal medication and 1 patient was on anti-viral medication concomitant with antibiotics.

CONCLUSION

Palifermin is safe and well tolerated and shows some effect in non-hematopoietic stem cell transplant pediatric patients with severe oral mucositis post-intensified chemotherapy or radiation therapy.

摘要

背景

严重口腔黏膜炎(OM)是化疗、放疗和干细胞移植后的不良事件之一,具有重大的临床和经济影响。严重OM的管理仍然具有挑战性。本研究旨在探讨帕利夫明对非移植儿科癌症患者黏膜炎的益处和临床影响。

方法

这是一项在沙特阿拉伯苏丹王子军事医疗城(PSMMC)进行的描述性回顾性研究,时间跨度为2016年至2020年。在此期间,对所有接受化疗并并发严重OM且需要使用帕利夫明(35个疗程)作为超说明书用药的儿科患者(<14岁)进行分析,以了解其临床人口统计学、主要诊断、使用的化疗药物、帕利夫明的有效性和耐受性。

结果

共有29例严重OM患者接受了35个帕利夫明疗程。他们均连续3天接受静脉注射60 mcg/kg/天的剂量。其中20%患有急性淋巴细胞白血病(ALL)。我们注意到,60%的严重OM在使用蒽环类药物后需要使用帕利夫明,而高剂量甲氨蝶呤会使40%的患者风险增加。只有25.7%的患者需要接受中位时长为5天的全胃肠外营养(TPN),54.3%的患者接受了中位时长为4天的阿片类药物治疗。20例患者(57.1%)使用了抗生素,4例患者使用了抗真菌药物,1例患者在使用抗生素的同时使用了抗病毒药物。

结论

帕利夫明安全且耐受性良好,在强化化疗或放疗后患有严重口腔黏膜炎的非造血干细胞移植儿科患者中显示出一定效果。

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