Background The 18-year-old age limit for inclusion in clinical trials constitutes a hurdle for adolescents and young adults (AYAs) with cancer. We analyzed the impact of this age barrier on the access of AYAs to cancer trials and novel therapies. Methods ClinicalTrials.gov was searched to identify all the trials including patients with 10 malignancies relevant for AYAs (January 2007 to July 2018). The trials were categorized as pediatric (patients <18 y), adult (≥18 y), and transitional (including adult and pediatric patients). Transitional trials with a lower limit between 12 and 18 years and an upper limit younger than 40 years were considered AYA-specific. Results Of 2764 identified trials, 2176 were included: 79% adult, 19% transitional, 2% pediatric. Five trials were AYA-specific. The proportion of academic trials was higher for transitional (69%; 288 of 421) than for adult trials (48%; 832 of 1718) (P < .0001). The total number of new trials increased over the years (156 in 2007; 228 in 2017); however, the number of transitional trials remained stable. The availability of trials increased with age, with a major increase at age 18 years: at age 17 years, 20% (442 of 2176) of trials were potentially accessible vs 95% (2075 of 2176) at 18 years. For trials investigating targeted therapies, this increase was 460% (197 trials available at age 17 years; 901 at 18 years) and for immunotherapies, 1200% (55 at age 17 years; 658 at 18 years). Conclusions AYAs have limited access to cancer trials and innovative therapies, with no improvement over the last decade. The 18-years-old age limit continues to be a major hurdle. Our findings are consistent with the internationally supported idea that age inclusion criteria in oncological trials should be changed.
Robust and applicable risk-stratifying genetic factors at diagnosis in pediatric T-cell acute lymphoblastic leukemia (T-ALL) are still lacking, and most protocols rely on measurable residual disease (MRD) assessment. In our study, we aimed to analyze the impact of NOTCH1, FBXW7, PTEN, and RAS mutations, the measurable residual disease (MRD) levels assessed by flow cytometry (FCM-MRD) and other reported risk factors in a Spanish cohort of pediatric T-ALL patients. We included 199 patients treated with SEHOP and PETHEMA consecutive protocols from 1998 to 2019. We observed a better outcome of patients included in the newest SEHOP-PETHEMA-2013 protocol compared to the previous SHOP-2005 cohort. FCM-MRD significantly predicted outcome in both protocols, but the impact at early and late time points differed between protocols. The impact of FCM-MRD at late time points was more evident in SEHOP-PETHEMA 2013, whereas in SHOP-2005 FCM-MRD was predictive of outcome at early time points. Genetics impact was different in SHOP-2005 and SEHOP-PETHEMA-2013 cohorts: NOTCH1 mutations impacted on overall survival only in the SEHOP-PETHEMA-2013 cohort, whereas homozygous deletions of CDKN2A/B had a significantly higher CIR in SHOP-2005 patients. We applied the clinical classification combining oncogenetics, WBC count and MRD levels at the end of induction as previously reported by the FRALLE group. Using this score, we identified different subgroups of patients with statistically different outcome in both Spanish cohorts. In SHOP-2005, the FRALLE classifier identified a subgroup of high-risk patients with poorer survival. In the newest protocol SEHOP-PETHEMA-2013, a very low-risk group of patients with excellent outcome and no relapses was detected, with borderline significance. Overall, FCM-MRD, WBC count and oncogenetics may refine the risk-stratification, helping to design tailored approaches for pediatric T-ALL patients.
Aneurysm of the inferior vena cava is a rare finding in the pediatric population. We report the case of a 5-month-old infant presenting with anemia, hypertension, and dehydration in the emergency room. A renal mass was found with ultrasound and MRI and a renal tumor was first considered. Histopathologic review of the surgical specimen led to the diagnosis of aneurysmal dilatation of the vena cava.
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