Top 5 Takeaways

1. High Adverse Event Rate: 91.4% of patients reported experiencing at least one adverse event during treatment with nifurtimox.
2. Demographic Concentration: Most patients were adults aged ≥18 years (91.8%) and Hispanic (93.2%).
3. Geographic Distribution: 34.2% of the patients were from California, the state with the highest number of treated patients.
4. Common Adverse Events: The most frequently reported adverse events were gastrointestinal, neurologic, and constitutional symptoms.
5. FDA Approval: Nifurtimox (Lampit) was approved by the FDA on August 6, 2020, and became commercially available in October 2020.

Original Article Author and Citation

Corresponding Author

Andrew Abbott, aabbott@cdc.gov

Suggested Citation

Abbott A, Montgomery SP, Chancey RJ. Characteristics and Adverse Events of Patients for Whom Nifurtimox Was Released Through CDC-Sponsored Investigational New Drug Program for Treatment of Chagas Disease — United States, 2001–2021. MMWR Morb Mortal Wkly Rep 2022;71:371–374. DOI: http://dx.doi.org/10.15585/mmwr.mm7110a2

Summary

This report summarizes the characteristics and adverse events of 336 patients treated with nifurtimox for Chagas disease under a CDC-sponsored Investigational New Drug (IND) program from 2001 to 2021. The majority of patients were adults and Hispanic, with a significant portion residing in California. A high rate of adverse events was reported, with gastrointestinal, neurologic, and constitutional symptoms being the most common. The FDA approved nifurtimox (Lampit) for commercial use in 2020, making it more accessible to healthcare providers.

Methods

Patient characteristics and adverse events were recorded by physicians who requested nifurtimox and monitored patients during and after treatment. Data were excluded for releases made under FDA individual IND authorizations and incomplete requests. Statistical significance was assessed using Fisher’s exact test, and analyses were performed using R and QGIS.

Discussion

The report provides the most comprehensive description of patients treated with nifurtimox and their adverse events during the 20-year period when the CDC was the sole provider. The findings highlight the importance of considering the frequency and profile of adverse events when prescribing nifurtimox. The high rate of adverse events, especially severe ones, underscores the need for careful patient counseling and monitoring.

Conclusion

Nifurtimox is now commercially available, improving access to treatment for Chagas disease. However, healthcare providers should be aware of the high frequency of adverse events and incorporate this information into patient counseling and treatment decisions. The CDC continues to offer diagnostic testing and teleconsultative services for Chagas disease.

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