دورية أكاديمية
Ruxolitinib for steroid-refractory graft versus host disease in pediatric HSCT: high response rate and manageable toxicity.
| العنوان: | Ruxolitinib for steroid-refractory graft versus host disease in pediatric HSCT: high response rate and manageable toxicity. |
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| المؤلفون: | Mozo Y; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Bueno D; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Sisinni L; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Fernández-Arroyo A; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Rosich B; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Martínez AP; Pediatric Hemato-Oncology Department, La Paz University Hospital, Madrid, Spain., Benítez-Carabante MI; Pediatric Hemato-Oncology Department, Hospital Universitari Vall d´Hebron, Barcelona, Spain., Alonso L; Pediatric Hemato-Oncology Department, Hospital Universitari Vall d´Hebron, Barcelona, Spain., Uría ML; Pediatric Hemato-Oncology Department, Hospital Universitari Vall d´Hebron, Barcelona, Spain., Heredia CD; Pediatric Hemato-Oncology Department, Hospital Universitari Vall d´Hebron, Barcelona, Spain., Mestre-Duran C; Traslational Research Group in Pediatric Hemato-Oncology, Transplantation and Cell Therapy, Hospital La Paz Institute for Health Research, Madrid, Spain., Pascual CFB; Traslational Research Group in Pediatric Hemato-Oncology, Transplantation and Cell Therapy, Hospital La Paz Institute for Health Research, Madrid, Spain., Torres J; Immunology Department, La Paz University Hospital, Madrid, Spain., Losantos I; Statistic Department, La Paz University Hospital, Madrid Spain., Escudero A; Translational Research in Pediatric Oncology, Hematopoietic Transplantation & Cell Therapy, Hospital La Paz Institute for Health Research (INGEMM-IdiPAZ), Institute of Medical and Molecular Genetics (INGEMM), La Paz University Hospital, Madrid, Spain., Ruz-Caracuel B; Translational Research in Pediatric Oncology, Hematopoietic Transplantation & Cell Therapy, Hospital La Paz Institute for Health Research (INGEMM-IdiPAZ), Institute of Medical and Molecular Genetics (INGEMM), La Paz University Hospital, Madrid, Spain. |
| المصدر: | Pediatric hematology and oncology [Pediatr Hematol Oncol] 2021 May; Vol. 38 (4), pp. 331-345. Date of Electronic Publication: 2021 Mar 04. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Informa Healthcare Country of Publication: England NLM ID: 8700164 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1521-0669 (Electronic) Linking ISSN: 08880018 NLM ISO Abbreviation: Pediatr Hematol Oncol Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2006- : London : Informa Healthcare Original Publication: Washington, DC : Hemisphere Pub. Corp., c1986- |
| مواضيع طبية MeSH: | Graft vs Host Disease/*drug therapy , Hematopoietic Stem Cell Transplantation/*adverse effects , Protein Kinase Inhibitors/*therapeutic use , Pyrazoles/*therapeutic use, Adolescent ; Child ; Child, Preschool ; Female ; Graft vs Host Disease/etiology ; Humans ; Infant ; Janus Kinases/antagonists & inhibitors ; Male ; Nitriles ; Protein Kinase Inhibitors/adverse effects ; Pyrazoles/adverse effects ; Pyrimidines ; Retrospective Studies ; Treatment Outcome |
| مستخلص: | Ruxolitinib, a selective Janus Kinase (JAK) 1/2 inhibitor, is a promising treatment for the steroid-refractory graft-vs-host disease (GvHD) after hematopoietic stem cell transplantation (HSCT). Most studies have been performed in the adult population showing efficacy against GvHD. In this retrospective study, we evaluated the outcomes of 19 children who received ruxolitinib for refractory acute or chronic GvHD (cGvHD) after HSCT from two Pediatric Hemato-Oncology Departments in Spain between March 2017 and December 2018. Patients received a median number of 4 (IQR 2) previous lines of treatment before starting ruxolitinib. The overall response rate in acute GvHD (aGvHD) and cGvHD was 87% and 91%, respectively. Complete response (CR) was observed in 37% of aGvHD and 8.3% of cGvHD. Remarkably, 43% and 40% of patients with steroid-refractory gastrointestinal aGvHD and lung cGvHD achieved CR. During ruxolitinib treatment, there were 36%, 31%, and 10% infections caused by viruses, bacteria, and fungi, respectively. Overall, four patients interrupted ruxolitinib due to infectious complications, hematological, and liver toxicity. The 2-year overall survival was 71.9% (CI 95% 58.6-85.2). Our experience supports the use of ruxolitinib as an effective treatment for steroid-refractory acute and cGvHD in children with a moderate toxicity profile. |
| فهرسة مساهمة: | Keywords: Graft versus host disease; hematopoietic stem cell transplantation; pediatric; ruxolitinib |
| المشرفين على المادة: | 0 (Nitriles) 0 (Protein Kinase Inhibitors) 0 (Pyrazoles) 0 (Pyrimidines) 82S8X8XX8H (ruxolitinib) EC 2.7.10.2 (Janus Kinases) |
| تواريخ الأحداث: | Date Created: 20210304 Date Completed: 20210610 Latest Revision: 20211204 |
| رمز التحديث: | 20221213 |
| DOI: | 10.1080/08880018.2020.1868637 |
| PMID: | 33661711 |
| قاعدة البيانات: | MEDLINE |
PDF full text from Publisher | تدمد: | 1521-0669 |
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| DOI: | 10.1080/08880018.2020.1868637 |