The challenges of clinical trials for adolescents and young adults with cancer
ABSTRACT In the United States, Europe, and Australia, and probably all countries of the world, older adolescents and young adults with cancer are under-represented in clinical trials of therapies that could improve their outcome. Simultaneously, the survival and mortality rates in these patients have mirrored the clinical trial accrual pattern, with little improvement compared with younger and older patients. This suggests that the relative lack of participation of adolescent and young adult patients in clinical trials has lessened their chances for as good an outcome as that enjoyed by patients in other age groups. Thus, increased availability of and participation in clinical trials is of paramount important if the current deficits in outcome in young adults and older adolescents are to be eliminated. Regardless of whether there is a causal relationship, the impact of low clinical trial activity on furthering our scientific knowledge and management of cancer during adolescence and early adulthood is detrimental.
- SourceAvailable from: Marilyn M Bui[Show abstract] [Hide abstract]
ABSTRACT: Background. Little is known about how cumulative chemotherapy delivery influences the poorer outcome observed in young adult (YA, 18-40 years) versus pediatric (<18 years) osteosarcoma patients. Here, we retrospectively examined differences in presentation, therapy, including cumulative chemotherapy dose, and outcome in YA and pediatric patients. Methods. We reviewed 111 cases of high-grade osteosarcoma at Moffitt Cancer Center between 1988 and 2012. Presentation factors, therapies, and survival were compared between YA and pediatric cohorts. Results. The cohorts were equivalent with respect to metastatic status, gender, tumor size, tumor site, and histological subtype. We found that the YA patients tended to have poorer histologic response to neoadjuvant chemotherapy measured by necrosis with 55% and 35% of pediatric versus YA patients responding favorably (P = 0.06). Only 39% of YA patients achieved the typical pediatric dose of methotrexate, doxorubicin, and cisplatin. These patients had a 3-year EFS of 76% (CI 53-100%) versus 47% (CI 26-69%; P = 0.09) in those who received less chemotherapy. Conclusion. Age continues to be a prognostic factor in osteosarcoma. Our study suggests that presentation factors are not associated with prognosis, while poorer response to chemotherapy and lower cumulative dose of chemotherapy delivered to YA patients may contribute to poorer outcomes.Sarcoma 04/2014; 2014:402509. DOI:10.1155/2014/402509
Article: Clinical trials.[Show abstract] [Hide abstract]
ABSTRACT: The spectrum of evidence imparted by the different clinical research designs ranges from ecological studies through observational epidemiological studies to randomized control trials (RCTs). This chapter addresses the definition of clinical research, the major aspects of clinical trials eg ethics, randomization, masking, recruitment and retention of subjects enrolled in a clinical trial, patients/subjects lost to follow-up during the trial etc. Although this chapter focuses on the weaknesses of clinical trials, it is emphasized that the randomized, placebocontrolled, double blind clinical trial is the design that yields the greatest level of scientific evidence. A researcher is in a gondola of a balloon that loses lift and lands in the middle of afield near a road. Of course, it looks like the balloon landed in the middle of nowhere. As the researcher ponders appropriate courses of action, another person wanders by. The researcher asks, ‘Where am I?’ The other person responds, ‘You are in the gondola of a balloon in the middle of a field.’ The researcher comments, ‘You must design clinical trials.’ ‘Well, that’ amazing, how did you know?’ ‘Your answer was correct and precise and totally useless.’Bulletin of the American College of Surgeons 05/2003; 88(5):43. DOI:10.1007/978-1-4020-8486-7_3