By Rebecca Berman, Janet Segall and Jean-Claude Bystryn, M.D. from The National Pemphigus Foundation and The Ronald O. Perelman Department of Dermatology, New York University School of Medicine, New York, NY. February 17, 1999

As many of you know, we recently conducted a survey of persons with pemphigus to find out 1) which treatments were most commonly used, 2) which appeared to be the most effective, and 3) which were most often associated with side effects. The survey was conducted by means of a questionnaire enclosed in the Fall 1998 issue of the National Pemphigus Foundation newsletter, the Quarterly. The questionnaire was also sent to all individuals who responded to a notice on the NPF website.A total of 110 responses were received. This number is impressive, taking into account the rarity of pemphigus. We thank all participants for their collaboration.

Most respondents had tried multiple agents. Overall, 8% of respondents tried one agent, 24% tried two, 28% tried three, 17% tried four, and 22% tried five or more of the drugs listed above. Currently, 33% of patients are using one medication, 42% are using two medications, 17% are using three medications, and 10% of patients are using four or more medications.

The agents used most commonly for the treatment of pemphigus were systemic steroids, topical steroids and Imuran, which were tried by 78%, 72% and 64% of respondents respectively. The least commonly used agents were Cyclosporin, Tetracycline and Methotrexate, which were tried by 8%, 13% and 14% of people respectively. The drugs that appeared to be the most beneficial from the respondents' perspectives were: systemic steroids, Imuran and topical steroids, which were described as helpful by 100%, 62% and 60% of patients who used the drugs. The least beneficial drugs seemed to be Tetracycline, Dapsone, and Methotrexate, whose use was discontinued because "it did not work" by 43%, 24% and 20% of patients treated with the drugs.

The drugs associated most often with side effects severe enough for treatment with that drug to be terminated were Cytoxan, Methotrexate and Imuran, which were discontinued by 38%, 33%, and 27% of users. To estimate the apparent relative effectiveness of the various drugs used to treat pemphigus, we calculated the ratio of respondents who believed the drug was useful to those who thought it did not work.

On that basis, the most effective drug was systemic steroids, with a ratio of 25. The ranking of the other drugs, based on their relative perceived effectiveness, were Imuran (ratio 5.1), topical steroids (ratio 5), Cytoxan (ratio 3.5), intralesional steroids (ratio 3.3), Methotrexate (ratio 2), Dapsone (ration 1.4), Cyclosporin (ratio 1.3), and Tetracycline (ratio 1.1). This survey was very helpful in collecting data from a large number of patients suffering from pemphigus.

However, it must be kept in mind that the reliability of the data is subject to question. One problem is that patients' evaluations of response to treatment is subjective, that in many cases treatment was given over a number of years and that memory of earlier treatment may not be completely accurate; and lastly, that several agents may be used concurrently so that the determination of which was effective is difficult. We again wish to thank all respondents for their time and effort. We hope you will be able to respond to additional questionnaires in the future.