Roche Holding AG (ROG)’s top-selling drug Rituxan effectively cured most cases of a rare and deadly skin disease in a patient study that suggests the medicine may replace steroids as the standard treatment.
In a trial among 22 people with severe pemphigus, a disorder marked by blisters on the skin and in the mouth and genitals, 59 percent were symptom-free more than six years after treatment with a single dose of Rituxan, researchers at France’s National Institute of Health and Medical Research in Rouen wrote in the journal Science Translational Medicine today. (March 6, 2013)
Pemphigus is usually treated with steroids, though relapses are common, requiring long-term treatment that can cause complications including diabetes, osteoporosis and severe infections. While the study tested Rituxan in patients already treated with steroids, the researchers expect results in about a year from a larger trial that compares the drug with steroids as a front-line therapy, said Philippe Musette, a professor of dermatology who led the research.
“In an autoimmune disease it’s really difficult to have complete remission,” Musette said in a telephone interview. “With one shot of rituximab we have a long-term result,” he said, using Rituxan’s generic name.
Rituxan, approved as a treatment for lymphoma, leukemia and rheumatoid arthritis, had sales of 6.7 billion Swiss francs ($7.1 billion) last year, making it Basel, Switzerland-based Roche’s biggest-selling product. The drug is sold as MabThera outside the U.S.
Roche has no plans to seek regulatory approval of Rituxan for the treatment of pemphigus, Daniel Grotzky, a company spokesman, said in an e-mail. The drug-maker is aware that some doctors prescribe the medicine for pemphigus, but doesn’t promote the so-called off-label use of any of its products, Grotzky said. Doctors in some countries, including the U.S., are allowed to prescribe drugs that are approved for one disease for any other disease.
In the trial, a single dose of Rituxan cleared the lesions caused by pemphigus in all but one of the patients in an average of 3 months. Seventeen patients relapsed, of whom nine received a second dose, with seven achieving complete remission — meaning they had no symptoms and required no further treatment. Two patients died of heart disease, who were also considered to be in complete remission.
Pemphigus is a so-called autoimmune disease in which the immune system attacks proteins needed to glue cells together. It strikes as many as seven people per million each year, mostly in middle-age or older.
Before it was treated with steroids, about 70 percent of people with the disease would die within a year, according to the Sacramento, California-based International Pemphigus and Pemphigoid Foundation.
]]>Schlüsselwörter
- Autoantikörper
- bullöse Autoimmundermatosen
- Pemphigus vulgaris
- Register
Zusammenfassung
Hintergrund: Rituxmab hat sich in kleinen Fallserien bei Patienten mit schwerem und/oder refraktärem Pemphigus als effektiv erwiesen. Bislang liegt jedoch keine systematische Untersuchung vor, die diese Beobachtung bestätigen könnte. Das Ziel dieser Studie war es, die Effektivität und Sicherheit von Rituxmab beim therapierefraktären Pemphigus anhand eines Registers systematisch zu ermitteln.Patienten und Methoden: Multizentrische, retrospektive Beobachtungsstudie an 36 Patienten mit schwerem Pemphigus vulgaris (n = 33) und Pemphigus foliaceus (n = 3), die vor dem 31. Sugust 2008 mit Rituxmab behandelt und zwischen Dezember2008 und Juni 2009 in ein nationales Beobachtungsregister eingeschlossen wurden.
Ergebnisse: Innerhalb einer durchschnittlichen Beobachtungsdauer von 11 (1-37) Monaten zeigten 21 (58%) Pemphiguspatienten ein komplettes, 13 (36%) ein partielles und 2 (6%) kein Ansprechen auf die Rituximab-Behandlung. Parallel hierzu kam es zur durchschnittlichen Verbesserung des subjektiven Wohlbefindens auf der visuellen Analogskala mit 34 Punkten (20-60) vor Behandlungsbeginn und 75 (40-95) bei der letzten Kontrollvisite. Bei 4 (11%) Patienten wurden schwerwiegende Nebenwirkungen beobachtet, u.a. eine schwere Infektion (3%). Schlussfolgerungen: Die systematischen Register erhobenen Daten weisen darauf hin, dass Rituxmab eine effektive und relativ sichere adjuvante Behandlungsoption für den refraktären Pemphigus ist. Kontrollierte prospektive Studien sind notwendig, um genauere Kenntnisse über die Effektivität und Sicherheit dieses Medikamentes zu gewinnen.
Summary:
Background: Rituxmab has been reported to be effective in various small case series of patients with severe and/or refractory pemphigus. However, nosystematic evaluation is available to corroborate this observation. The aim of this study was to systematically determine efficacy and safety of rituxmab in treatment-resistent pemphigus.
JDDG: Journal der Deutschen Dermatologischen Gesellschaft
]]>Variété de pemphigus vulgaire se caractérisant par la présence de bulles qui finissent par confluer (se rejoindre) et se rompre, créant ainsi de vastes surfaces laissant la peau à vif qui suinte et qui devient très douloureuse.
Généralités
Le pemphigus vulgaire est une variété de pemphigus vrai (groupe des maladies cutanées se caractérisant par l’importance des bulles et l’évolution péjorative entre autres) qui survient à l’âge moyen de la vie et qui se caractérise par l’apparition de bulles de taille plus ou moins importantes, se remplissant d’un liquide de coloration jaune citrin, indolore, sur l’ensemble du corps avec une prédominance aux plis et sur les muqueuses (couche de cellules recouvrant l’intérieur des organes creux en contact avec l’air) essentiellement celles de la bouche.
Historique
Le français Brocq, en 1919, a étudié cette dermatose (maladie de peau) grave.
Évolution
Étant donné la gravité de l’atteinte générale survenant au cours de cette pathologie, l’évolution est souvent péjorative en quelques semaines.
From: Acta medica Iranica
]]>Article: http://www.vetsmall.theclinics.com/article/S0195-5616(13)00009-0/abstract
Pictures: http://www.sciencedirect.com/science/article/pii/S0195561613000090
]]>Recently, the IPPF has welcomed two new Peer Health Coaches to our team, Mei Ling Moore (Los Angeles, CA) and Gloria Gutierrez (Orlando, FL). They both have been providing support for our community members for quite some time so it seemed only natural for them to volunteer as Peer Health Coaches. Both are compassionate listeners who actively participate on the IPPF website and Facebook page, communicate well with those that need support, provide relevant resources designed to improve patient/caregiver issues and make a difference in people’s lives by building long-lasting relationships.
I had the honor of seeing them in action recently at our annual Patient Conference in San Francisco and was amazed at how well they both provided confidence and hope to everyone they spoke with.
Please join me in welcoming Mei Ling and Gloria and feel free to reach out to them for peer advice.
Remember, you always have a “Coach” in your corner!
]]>Abbreviations:
Dsg, Desmoglein; GFP, Green fluorescent protein; TRAIL, tumor necrosis factor -related apoptosis-inducing ligand; HNSCC, head and neck squamous cell carcinoma; scFvs, single chain variable fragment antibodies; HA, hemaggultinin
]]>Aims: To evaluate the patients with pemphigus vulgaris on treatment in respect of osteoporosis and to compare the frequency of osteoporosis in these patients with the healthy ones.
Methods: The study consisted of 40 patients with pemphigus vulgaris and 34 healthy controls. Bone mineral density measurements were obtained by dual- energy X-ray absorptiometry. Blood serum, bone parameters, and biochemical hormonal measurements were examined in both groups.
Results: When the bone mineral density values of patients with pemphigus vulgaris were compared with those of the control group, there was no significant difference between hip bone mineral density values, while lumbar region T and Z scores were found significantly low in the patient group (p = 0.034 and p = 0.006, respectively). Osteoporosis, osteopenia, and normal dual-energy X-ray absorptiometry rates in the patient group were found to be 32.5%, 32.5%, and 35%, respectively. These rates were found to be 18%, 23%, and 59% in control group, respectively. There were more fractures in the patient group and the difference was statistically significant (p = 0.004).
Conclusion: An increase in osteoporosis frequency and secondary fracture to osteoporosis in the patients with pemphigus vulgaris was detected.
Full acticle can be viewed at: Indian Journal of Dermatology
]]>Adjuvant therapeutic methods are employed when pemphigus vulgaris (PV) fails to be controlled by conventional corticosteroid treatment. Objective: The efficacy of double filtration plasmapheresis (DFPP) was investigated in a PV patient with severe, refractory mucosal disease.
Methods
A total of 3 DFPP cycles, each cycle consisting of 5 double filtration sessions conducted on alternate days was completed.
Results
DFPP provided immediate clinical relief of symptoms as well as a significant decrease in anti-desmoglein antibody levels and allowed for a much lower corticosteroid dose.
Conclusion
DFPP was an effective and safe adjuvant therapy in our patient with PV and it offers a valid treatment option in PV patients with recalcitrant disease.
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