The challenges of clinical trials for adolescents and young adults with cancer. Pediatr Blood Cancer

Pediatric Oncology Unit, Istituto Nazionale Tumori, Milan, Italy.
Pediatric Blood & Cancer (Impact Factor: 2.39). 05/2008; 50(5 Suppl):1101-4. DOI: 10.1002/pbc.21459
Source: PubMed


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.

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    • "This may be due to presentation factors, such as late presentation due to patient denial, primary care physicians having a low suspicion of malignancy in a young adult, or inadequate access to care. Therapy factors such as poor clinical trial participation, lack of a care system focused on the needs of the YA patient, and poor adherence to therapy may also contribute to the overall worse outcome [20] [21] [22] [23] [24]. It is known that adherence to a planned chemotherapy regimen impacts survival, and modifying the regimen in osteosarcoma has been associated with poorer local recurrence-free survival [25]. "
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    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.
    Full-text · Article · Apr 2014 · Sarcoma
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    • "Adolescent and young adults (AYA) (18–40 years old) typically fare worse than both the younger and older cohorts of patients for a given histologic diagnosis.100–102 This is thought to arise from a multitude of factors including location of care, patient education, poor clinical trial participation, and a lack of a care system focused on the needs of this patient group.103–107 In addition to osteosarcoma and Ewing sarcoma, sarcomas with a peak incidence in the AYA population include synovial sarcoma and Malignant Peripheral Nerve Sheath Tumor (MPNST). "
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    ABSTRACT: Sarcomas are cancers arising from the mesenchymal layer that affect children, adolescents, young adults, and adults. Although most sarcomas are localized, many display a remarkable predilection for metastasis to the lungs, liver, bones, subcutaneous tissue, and lymph nodes. Additionally, many sarcoma patients presenting initially with localized disease may relapse at metastatic sites. While localized sarcomas can often be cured through surgery and often radiation, controversies exist over optimal management of patients with metastatic sarcoma. Combinations of chemotherapy are the most effective in many settings, and many promising new agents are under active investigation or are being explored in preclinical models. Metastatic sarcomas are excellent candidates for novel approaches with additional agents as they have demonstrated chemosensitivity and affect a portion of the population that is motivated toward curative therapy. In this paper, we provide an overview on the common sarcomas of childhood (rhabdomyosarcoma), adolescence, and young adults (osteosarcoma, Ewing sarcoma, synovial sarcoma, and malignant peripheral nerve sheath tumor) and older adults (leiomyosarcoma, liposarcoma, and undifferentiated high grade sarcoma) in terms of the epidemiology, current therapy, promising therapeutic directions and outcome with a focus on metastatic disease. Potential advances in terms of promising therapy and biologic insights may lead to more effective and safer therapies; however, more clinical trials and research are needed for patients with metastatic sarcoma.
    Full-text · Article · May 2013 · Clinical Epidemiology
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