And orphan diseases

Orphan disease cancer

An important trial reported in this issue of Annals of Oncology builds upon this foundation and on the promising activity of paclitaxel in phase II trials. The randomised comparison of the standard three-weekly doxorubicin (60 mg/m2) plus cisplatin (50 mg/m2) with doxorubicin (50 mg/m2) plus paclitaxel (150 mg/m2 as a 24-h infusion) plus filgrastim was performed in an exemplary manner, based upon sound principles of clinical trial design. The randomisation yielded comparable groups of patients, and the selection of patients represented the population of endometrial cancer patients. Despite promising results from preliminary trials and the activity of paclitaxel in a phase III trial , the chemotherapy regimens did not differ significantly in terms of activity and toxicity. While this may seem disappointing, at least two points are illustrated: (i) promising observations in phase II trials may be due to patient selection bias and need not be confirmed in randomised trials; and (ii) cisplatin or carboplatin may be essential in the therapy of endometrial cancer. The latter hypothesis is supported by two very recent trials [, ].

A more general issue, however, also deserves a mention: certain neoplasias that are much rarer than endometrial cancer have been far better investigated. For instance, only 8000 cases of Hodgkin's disease are diagnosed annually in the United States, and a similar number is expected for the European Union; a substantial majority of these patients can be cured by chemotherapy and/or radiotherapy, such that ∼1300 patients a year die from Hodgkin's disease in the United States . A long series of trials organised by many collaborative groups in different countries have optimised chemotherapy and radiotherapy to the current state of the art, which allows the majority of patients, even with advanced stage Hodgkin's disease, to be cured. By comparison, endometrial cancer appears to be a poorly investigated orphan disease indeed, despite its frequent occurrence. A substantial decrease in the incidence of endometrial cancer is unlikely in the next few years, and early detection methods have not been proven to have a major impact on mortality . Thus, our efforts should be focused on improving all treatment modalities for endometrial cancer. Such progress will have to begin in the minds of those who care for patients with endometrial cancer. This tumour is neither rarer nor less interesting than other neoplasias that have been studied successfully. In addition to elucidating the molecular events that lead to and characterise endometrial cancer, the oncology community is obliged to set up and complete clinical trials of endometrial cancer surgery, radiation therapy and medical therapy. At present, the North American Gynecologic Oncology Group is leading the way, but despite regulatory agencies increasingly threatening clinical research, similar contributions will have to be made in Europe.

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