An EIN member asked for suggestions for obtaining clofazimine for a patient with progressive Mycobacterium avium-intracellulare (MAI) disease who had previously been treated with standard first- and second-line medications.

Several members noted that the drug is no longer commercially available in the United States, but can be obtained for free through the Food and Drug Administration under an investigational new drug ( IND) application. Some members added that institutional review board (IRB) approval will be required. A letter from the manufacturer, Novartis Pharmaceuticals Corporation, with more information and contact details can be found at FDA’s drug shortages website.

A member in Florida who sees a large number of patients with MAI offers the following suggestions: “When progression occurs on treatment, as defined by worsening CT chest and symptoms (cough, shortness of breath, hemoptysis, weight loss, etc.), look for the following: 1) non-compliance with the regimen; 2) secondary pathogens such as rapidly growing mycobacteria (RGM), Pseudomonas, MRSA, or Aspergillus (consider bronchoalveolar lavage), or 3) truly resistant MAI. If all else fails, for extremely resistant cases you may want to consider gamma interferon for eight weeks or lung resection of large cavity or isolated lobe involvement.”

However, “This discussion misses some very important points,” added a member in California. “First, there is no evidence that conventional in vitro susceptibility test results with clofazimine correlate with clinical outcomes, and at least some evidence that indicates they do not (see, e.g., J Infect Dis. 1996;173:677-83). Furthermore, there is no evidence that clofazimine contributes to a favorable result in treating patients with Mycobacterium avium complex (MAC) infection (see, e.g., Int Conf AIDS 1992; 8:B101, abstract no. PoB 3087; Eur J Clin Microbiol Infect Dis. 1999;18:16-22; AIDS 1997;11:311-7). (It must be noted that these deal with disseminated MAC in AIDS and were likely caused by M. avium, while the patient in question has pulmonary infection and is more likely to be infected with M. intracellulare.)”

“Finally,” the member concluded, “before you go through all the trouble of getting the drug, you may wish to discuss with the patient the almost inevitable skin discoloration that occurs with prolonged use. Many patients will opt out at that point.”

A joint project of IDSA and the Pediatric Infectious Diseases Society (PIDS) and supported by funding from the Centers for Disease Control and Prevention.

EIN tracks emerging infectious diseases and keeps the public-health community up to date with issues that are currently affecting or may soon affect members’ clinical practices.The Network provides a great opportunity for members to share knowledge quickly across large geographical distances. Both IDSA and PIDS members are eligible to join. The EIN listserve allows members to discuss new disease trends and difficult cases. Click here for more information or to join EIN.

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