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A phase II trial of nifurtimox combined with topotecan and cyclophosphamide for refractory or relapsed neuroblastoma and medulloblastoma.

Author: BeeravallyNagulapallyAbhinav, BergendahlGenevieve, BrownValerie I, DykemaKarl, EslinDon, FergusonWilliam, FluchelMark, GinnKevin, HannaGina, HigginsTimothy, IsakoffMichael S, KravekaJacqueline, LewisElizabeth, MitchellDeanna, RawwasJawhar, RobertsWilliam, Saulnier ShollerGiselle L, WadaRandal K, ZagePeter E

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Overview

This clinical trial investigated the efficacy of a combination therapy including nifurtimox (Nfx), topotecan, and cyclophosphamide in children with relapsed/refractory (R/R) neuroblastoma (NB) and medulloblastoma (MB). The combination showed a significant response rate and disease stabilization, suggesting it as a potential treatment option for these challenging cancers.
Paper Details　
Original Abstract of the Article :
Children with relapsed/refractory (R/R) neuroblastoma (NB) and medulloblastoma (MB) have poor outcomes. We evaluated the efficacy of nifurtimox (Nfx) in a clinical trial for children with R/R NB and MB. Subjects were divided into three strata: first relapse NB, multiply R/R NB, and R/R MB. All ...See full text at original site
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引用元：
https://doi.org/10.1002/ijc.34569

データ提供：米国国立医学図書館（NLM）

A New Oasis in the Desert of Refractory Neuroblastoma and Medulloblastoma: Exploring Nifurtimox Combination Therapy

The treatment of relapsed or refractory (R/R) neuroblastoma and medulloblastoma, aggressive pediatric cancers, continues to be a formidable challenge. This study investigates the efficacy and safety of a novel combination therapy involving nifurtimox (Nfx), topotecan, and cyclophosphamide in children with R/R neuroblastoma and medulloblastoma. The researchers conducted a phase II clinical trial involving 112 eligible patients, evaluating the response rates and safety profile of the combination therapy.

Navigating the Challenging Landscape of Pediatric Cancers: A Search for Effective Therapies

The study found encouraging results, demonstrating a significant response rate in patients with first relapse neuroblastoma (53.9%) and a high total benefit rate in all three patient strata (first relapse neuroblastoma, multiply R/R neuroblastoma, and R/R medulloblastoma). The combination therapy was generally well tolerated, with the most common side effects being bone marrow suppression and reversible neurological complications.

A Glimmer of Hope in the Desert: Exploring the Potential of Nifurtimox Combination Therapy

This study offers a glimmer of hope in the fight against R/R neuroblastoma and medulloblastoma. The promising response rates and manageable side effects suggest that the combination therapy involving Nfx, topotecan, and cyclophosphamide may represent a valuable treatment option for these challenging cancers. Further research is warranted to confirm these findings and explore the optimal use of this therapy in the treatment of pediatric cancers.

Dr.Camel's Conclusion

The desert of childhood cancer is unforgiving, but this study offers a potential oasis in the form of a promising new combination therapy. While more research is needed, the results provide a glimmer of hope for children battling these aggressive cancers. It's a testament to the relentless spirit of medical innovation, which continues to seek new ways to overcome the challenges posed by devastating diseases.

Date :
  1. Date Completed 2023-07-17
  2. Date Revised 2023-11-16
Further Info :

Pubmed ID

37246577

DOI: Digital Object Identifier

10.1002/ijc.34569

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Article Analysis
・Side Effects of nifurtimox
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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