Extrait

Background and study aims Polyarteritis nodosa (PAN) is a serious inflammatory blood vessel disease (vasculitis) in children. It is the third most common vasculitis in childhood and presents with fever, arthritis, skin rash and stomach pain, amongst other symptoms. Following the introduction of steroid treatment in the 1950s, death rates fell from 100% to 50%. The introduction of cyclophosphamide (CYC) in the 1970s resulted in further significant reductions in death rate. Surprisingly there have been no further advances in treatment since then, and no clinical studies ever performed in children with PAN. Currently, the death rate is between 3 and 10%, although this comes at a cost: 25 to 50% experience a serious side effect of CYC including severe infection, nausea and hair loss, and increased risk of late infertility and malignancy. Clinical studies of alternative drugs to replace CYC are therefore important. The drug mycophenolate mofetil (MMF) is believed to be a safer treatment option than CYC. The main aim of the study is to find out how safe and effective MMF is for the treatment of PAN, compared to the standard drug CYC. Who can participate? Children aged 4 to 18 years diagnosed with PAN can take part. What does the study involve? Participants will be randomly allocated to one of two groups: 3-6 months (12-24 weeks) treatment with either MMF or CYC. This is followed by 12-15 months (48-60 weeks) azathioprine plus prednisolone for all. The study ends after 18 months (72 weeks) treatment. What are the possible benefits and risks of participating? Not provided at time of registration. Where is the study run from? The study takes place in various vasculitis centres across Europe: 1. Great Ormond Street Hospital, UK 2. Sheffield Children's Hospital, UK 3. University Hospital Bristol, UK 4. Royal Manchester Children's Hospital, UK 5. Royal Hospital for Sick Children, Glasgow, UK 6. Birmingham Children's Hospital, UK 7. Nottingham University Hospital, UK 8. Norfolk and Norwich University Hospital NHS Trust, UK 9. Royal Victoria Hospital, Newcastle, UK 10. Alder Hey Children's NHS Foundation Trust, UK 11. Universitair Ziekenhuis Gent, Belgium 12. University Hospital Centre Zagreb, University School of Medicine, Croatia 13. Charles University in Prague and General University Hospital, Czech Republic 14. Klinikum Bad Bramstedt, Germany 15. Charite University Hospital Berlin, Germany 16. Helios Kliniken Berlin, Germany 17. University Medical Center Freiburg, Germany 18. Kinderklinik Garmisch-Partenkirchen gGmbH, Germany 19. Athens Medical School, University of Athens, Greece 20. IRCCS Istituto G. Gaslini, Italy 21. Azienda Ospedaliero-Universitaria Meyer, Italy 22. Ospedale Santa Chiara, Università di Pisa, Italy 23. Ospedale Pediatrico Bambin Gesu‘, Italy 24. Wilhelmina Kinderziekenhuis, Netherlands 25. Medical University of Silesia, Poland 26. Wojewódzki Specjalistyczny Szpital Dziecięcy św. Ludwika w Krakowie, Poland 27. Hospital Sao Joao, Portugal 28. Hospital Santa Maria, Portugal 29. University Children's Hospital, University Medical Centre Ljubljana, Slovenia 30. Hospital Ramon y Cajal, Spain 31. Hospital Universitario La Fe, Spain 32. Hospital Sant Joan de Deu, Universitat de Barcelona, Spain 33. Kanuni Sultan Süleyman Education and Research Hospital, Turkey 34. The Queen Silvia Children's Hospital, Sweden 35. Gulhane Military Medical Academy School of Medicine, Turkey 36. Hacettepe University Children's Hospital, Turkey When is the study starting and how long is it expected to run for? November 2014 to June 2019. Who is funding the study? Arthritis Research UK. Who is the main contact? Mr Ben Hardwick [email protected]

Critère d'inclusion

• Topic: Children; Subtopic: All Diagnoses; Disease: All Diseases

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