Recent articles in the medical literature will be of interest to our readers. A Letter to the Editor in the April 2008 issue of the Journal of Ophthalmology discussed a preliminary study from Italy of intravenous immunoglobulins (IVIg) for the treatment of Mucous Membrane Pemphigoid (MMP). In it, 6 patients with severe MMP were treated with IVIg for 8 to 20 months.
All responded to this treatment with healing of mouth lesions and stoppage of eye inflammation and scarring. As the treatments went on and symptoms decreased, steroids and immunosuppressants were slowly reduced and the time between IVIg cycles was lengthened. There were side effects but all were mild. They will continue to follow these patients as IVIg is discontinued. The authors also called for a larger study to confirm their findings. This study seems to indicate IVIg works in MMP as it has been found to work in pemphigus patients.
The journey for patients and caregivers trying to get diagnosed, educated and treated for pemphigus or pemphigoid is difficult. The June 14, 2008 issue of The Lancet has articles, and an editorial on rare diseases and orphan drugs. One article indicates, though not by name, that pemphigus and pemphigoid are probably best defined as ultra-rare. Titles in the issue included:
- Making rare diseases a public-health and research priority;
- Why rare diseases are an important medical and social issue;
- Empowerment of patients: lessons from the rare diseases community; and
- Does orphan drug legislation really answer the needs of patients.
As you can see these are all topics that involve the pemphigus and pemphigoid community. The fact that a major medical journal is committing so much space to these problems is good news. However, progress is slow and costly.
The National Organization of Rare Disorders (NORD) is discussed. The IPPF is a member of this group. Eurordis is a similar organization for Europeans with rare diseases and Orphanet is a resource for rare disease web sites. The articles show that patients and their organizations like the IPPF have moved the general medical community to some action. They have also moved legislative and pharmaceutical companies to pursue orphan drug legislation. Because the accumulative number of people with rare disease is substantial, it is pointed out that treating and helping these people benefits the whole community. However much remains to be done.
In conclusion, I would like to quote from the lead editorial:
There are encouraging signs that rare diseases are being included in public-health plans, and that more research is being directed in finding the causes of and treatments for at least some of these conditions. Patients with rare diseases have the right to first class medical knowledge, rapid diagnosis, research, and treatment [emphasis added]. In reality, though, their lives are often blighted by medical ignorance, lack of professional advice, delayed diagnosis, and no treatment options at all. Patients should not have to depend on their families to find potential treatments for rare diseases.
