التفاصيل البيبلوغرافية
| العنوان: |
Busulfan and Subsequent Malignancy: An Evidence-Based Risk Assessment |
| المؤلفون: |
Long-Boyle, Janel, Kohn, Donald, Shah, Ami, Spencer, Sueli Marques, Sevilla, Julian, Booth, Claire, López-Lorenzo, Jose Luis, Nicoletti, Eileen, Shah, Arpita, Reatz, Meredith, Matos, Joana, Schwartz, Jonathan |
| بيانات النشر: |
Authorea, Inc. |
| سنة النشر: |
2023 |
| المجموعة: |
The Winnower (via CrossRef) |
| الوصف: |
The incidence of secondary malignancies associated with busulfan exposure is considered low, but has been poorly characterized. Because this alkylating agent is increasingly utilized as conditioning prior to gene therapy in non-malignant hematologic and related disorders, more precise characterization of busulfan’s potential contribution to subsequent malignant risk is warranted. We conducted a literature-based assessment of busulfan and subsequent late effects, with emphasis on secondary malignancies, identifying publications via PubMed searches and selecting those reporting at least 3 years of follow-up. We identified 8 pediatric and 13 adult publications describing long-term follow-up in 570 pediatric and 2,076 adult hematopoietic cell transplant (HCT) recipients. Secondary malignancies were reported in 0.5% of pediatric HCT recipients, with no cases of myelodysplastic syndrome (MDS) or acute myelocytic leukemia (AML). Fatal secondary malignancies were reported in 0.8% of 1887 evaluable adult HCT recipients, and an overall incidence of secondary malignancies of 4.8% was reported in a subset of 389 evaluable adult patients. We also reviewed long-term results from 8 publications evaluating lentiviral- and human promotor-based HSC-targeted gene therapy in 215 patients with non-malignant conditions, in which busulfan/treosulfan monotherapy or busulfan/fludarabine was the only conditioning. Two malignancies were reported in patients with Sickle Cell Disease (SCD), one of which was potentially busulfan-related. No additional malignancies were reported in 173 patients with follow-up of 5-12 years. The incidence of busulfan-related secondary malignancies is low, and likely to be substantially less than 1% in pediatric transplant recipients, especially those receiving busulfan monotherapy for non-malignant conditions other than SCD. |
| نوع الوثيقة: |
other/unknown material |
| اللغة: |
unknown |
| DOI: |
10.22541/au.169259765.51803663/v1 |
| الإتاحة: |
https://doi.org/10.22541/au.169259765.51803663/v1Test |
| رقم الانضمام: |
edsbas.F73B8F4 |
| قاعدة البيانات: |
BASE |
