Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations
| العنوان: | Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations |
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| المؤلفون: | Richa Jain, Prateek Bhatia, Rekha Hans, Deepak Bansal, Bhagwant Rai Mittal, Amita Trehan, Neelam Marwaha, Sidharth Totadri, Rakesh Kapoor, Ratti Ram Sharma, Nandita Kakkar, Neelam Varma, Akshay Kumar Saxena, Radhika Srinivasan, Arvind Rajwanshi, Prema Menon, Ram Samujh |
| المصدر: | Pediatric Blood & Cancer. 67 |
| بيانات النشر: | Wiley, 2020. |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | Male, Oncology, Melphalan, medicine.medical_specialty, Transplantation Conditioning, Transplantation, Autologous, Cryopreservation, Neuroblastoma, 03 medical and health sciences, 0302 clinical medicine, Interquartile range, Internal medicine, medicine, Humans, High risk neuroblastoma, Retrospective Studies, Radiotherapy, business.industry, Plerixafor, Hematopoietic Stem Cell Transplantation, Hematology, Prognosis, medicine.disease, Combined Modality Therapy, Survival Rate, Apheresis, Child, Preschool, 030220 oncology & carcinogenesis, Pediatrics, Perinatology and Child Health, Female, Stem cell, business, Follow-Up Studies, 030215 immunology, medicine.drug |
| الوصف: | Background The majority of patients in low- and middle-income countries (LMIC) are unable to receive optimal therapy, including autologous stem cell transplant (ASCT) for high-risk neuroblastoma. Management is intensive and multidisciplinary; survival is often poor. We report a single-center outcome of high-risk neuroblastoma, with adaptations optimized for LMIC. Procedure The study was retrospective. Patients were treated on the backbone of the high-risk neuroblastoma study-1 of SIOP-Europe (HR-NBL1/SIOPEN) protocol with ASCT. Adaptations incorporated to decrease cost, requirement for inpatient admission, infections, and faster engraftment included (a) optional outpatient administration for rapid-COJEC, (b) two sessions of stem-cell apheresis, (c) storing stem cells at 2-6°C without cryopreservation for up to 7 days, (d) no central lines, (e) no antibacterial/antifungal/antiviral prophylaxis, (f) omitting formal assessment of cardiac/renal/pulmonary functions before ASCT, and (g) administration of pegylated granulocyte colony-stimulating factor on Day +4. Results Over 5 years 9 months, 35 patients with high-risk neuroblastoma were treated. Rapid-COJEC was administered over a median duration of 80 days (interquartile range: 77, 83). Conditioning regimen included melphalan (n = 7), oral busulfan-melphalan (Bu/Mel; n = 6), or intravenous Bu/Mel (n = 22). The median viability of stem cells stored for 6 days (n = 28) was 93% (range: 88-99). Two (5.7%) patients had ASCT-related mortality. The 3-year overall and event-free survival was 41% and 39%, respectively. A relapse occurred in 20 (57%) patients. Treatment abandonment was observed in one (3%) patient. Conclusions Administration of therapy in a disciplined time frame along with low-cost adaptations enables to manage high-risk neuroblastoma with low abandonment and an encouraging survival in LMIC. Stem cells can be stored safely without cryopreservation for up to 7 days. |
| تدمد: | 1545-5017 1545-5009 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::643b2e8da0f0cae684ad475150cd598fTest https://doi.org/10.1002/pbc.28273Test |
| حقوق: | CLOSED |
| رقم الانضمام: | edsair.doi.dedup.....643b2e8da0f0cae684ad475150cd598f |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15455017 15455009 |
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