Feasibility of randomised clinical trials in rare diseases: the case of polycythemia vera

Leukemia & Lymphoma
Roberto MarchioliGianni Tognoni

Abstract

Although it has long been recognised as the only reliable instrument for producing scientific evidence on the benefit/risk profile of therapeutic interventions, the technology of randomised clinical trials (RCT) is far from being the backbone of medical knowledge. Randomised clinical trials in polycythemia vera have been carried out when their methodology was being built up and, therefore, was rather unsatisfactory. Now we have aggressive cytoreductive treatments with chemotherapeutic agents loaded with doubts on long-term safety, while phlebotomy and preventive antiplatelet therapy are left to personal preferences because of debatable results of old, low-power clinical trials. A complex profile of uncertainties requires a simple, but articulated strategy of care and research to allow at the same time a reasonable transfer of the best available validated knowledge and a timely investigation of the most relevant questions. Without doubts, multi-country, collaborative RCTs is the key (not isolated or abstract) element of the current scenario. The declared background hypothesis is the willingness of a medical caring community of being, at the same time and with the same patients, a research community. The trial design comes in as ...Continue Reading

References

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Citations

Mar 1, 1997·Blood Reviews·A TefferiM N Silverstein
Oct 8, 2005·Seminars in Hematology·Roberto MarchioliTiziano Barbui
Feb 16, 2005·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Roberto MarchioliTiziano Barbui
Nov 25, 2003·Hematology·Jerry L SpivakMonia Marchetti

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