Randomized trials in rare tumors.

1986 ◽  
Vol 4 (2) ◽  
pp. 262-263
Author(s):  
G A Omura
1985 ◽  
Vol 3 (9) ◽  
pp. 1163-1165
Author(s):  
J F Holland

Author(s):  
Jonathan A. Ledermann ◽  
Isabelle Ray-Coquard

More than 50% of all gynecologic cancers can be classified as rare tumors (defined as an incidence of fewer than six per 100,000). Improved understanding of the molecular pathogenesis of tumors increases the proportion of rare tumors and creates challenges in optimizing the design of clinical trials. Novel trial designs are needed to take forward the development of new treatments in rare tumors. This requires international partnerships, harmonization of treatment, and collaboration to overcome the regulatory barriers to conducting international trials. Although randomized trials can be done in many tumor types, there are some for which conducting even single-arm studies may be challenging. For these tumors, robust collection of data through national and/or international registries could lead through audit to improvements in the treatment of rare tumors.


1993 ◽  
Vol 69 (02) ◽  
pp. 115-118 ◽  
Author(s):  
Kathelijne Peerlinck ◽  
Jef Arnout ◽  
Jean Guy Gilles ◽  
Jean-Marie Saint-Remy ◽  
Jos Vermylen

SummaryIn May 1990, 218 patients with haemophilia A regularly attending the Leuven Haemophilia Center were randomly assigned to a group receiving either of two newly introduced factor VIII concentrates: factor VIII-P, an intermediate purity pasteurized concentrate, or factor VIII-SD, a high purity concentrate treated with solvent-detergent for viral inactivation.Patients were followed from May 1990 until October 1991. Between August 1991 and October 1991 a clinically important factor VIII inhibitor was detected in five out of the 109 patients receiving factor VIII-P while none of the 109 patients receiving factor VIII-SD developed such antibodies. All patients acquiring an inhibitor had previously been clinically tolerant to transfused factor VIII with 200 to more than 1,000 days of exposure to factor VIII prior to May 1990. Patients with inhibitors were transfused daily with 30 U factor VIII-SD per kg body weight, which was associated with a gradual decline of the inhibitor level. In all patients the antibodies were relatively slow-acting and predominantly directed towards the light chain of factor VIII.This study demonstrates a higher than expected incidence of factor VIII inhibitors associated with the use of a specific factor VIII concentrate in multitransfused haemophilia A patients. It indicates the usefulness of evaluating newly introduced concentrates in prospective, randomized trials.


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