Confirmation of Bevacizumab Activity, and Maintenance of Efficacy in Retreatment After Subsequent Relapse, in Pediatric Low-grade Glioma.

BACKGROUND: Management of low-grade gliomas (LGG) can be a challenge, particularly when not resectable and refractory or recurrent following standard treatments. We undertook a retrospective analysis of 2 institutions' experiences treating children for refractory or progressive LGG with bevacizumab-based therapy (BBT). PROCEDURE: Inclusion criteria were patients younger than 18 years of age who had previously failed one or more lines of therapy. Treatment was intravenous bevacizumab 10 mg/kg and intravenous irinotecan 125 to 150 mg/m2 every 2 weeks. RESULTS: Sixteen children (median age of 8.6 y), 5 with neurofibromatosis type 1 and 8 with disseminated disease were treated between 2009 and 2013. Median duration of treatment was 12 months (range, 3 to 45 mo). Seven patients (44%) showed clinical improvement (3 patients within a month) and 8 patients (50%) remained clinically stable during BBT. Imaging studies showed 3 (19%) had a partial response, 11 (69%) stable disease, and 2 (12%) had progressive disease. Four patients had progressive disease after stopping BBT (median duration of 5 mo). Three of these 4 were able to be retreated with BBT and all achieved an objective response. Treatment was well tolerated with no grade 3 or 4 toxicities related to bevacizumab. Irinotecan was discontinued in 4 patients because of grade 2-3 toxicities. CONCLUSIONS: We conclude that BBT is well tolerated and led to disease control in patients with refractory or recurrent cases of LGG. Retreatment with BBT led to disease control in most of these cases. Larger, prospective studies are warranted to confirm these results.

Research with vulnerable families caring for children with life-limiting conditions.

Methodological challenges associated with sensitive research, such as research with vulnerable families, have been well described, but there are few examples of how such challenges have been addressed in specific projects. To help address this gap, we describe how we designed and conducted a qualitative study of 91 members of 29 families caring for children or young people with life-limiting conditions. Although some issues associated with researching sensitive topics were anticipated, others were encountered that were not expected. We discuss insights gained from this study in relation to factors that influence effectiveness of recruitment, the quality of data collection, and the safety of participants and researchers.

Understanding medication adherence in pediatric acute lymphoblastic leukemia: a review.

Significant numbers of children and adolescents with acute lymphoblastic leukemia (ALL) do not adequately adhere to their treatment regimen. Failure to take the appropriate amount of prescribed medication may result in disease relapse. Although a number of research studies have sought to identify the factors associated with medication nonadherence in this group, no systematic study has sought to evaluate the efficacy of intervention packages in improving adherence. The aim of the current paper is to provide an overview of the research investigating treatment adherence in ALL patients and to identify the relevant risk factors associated with reduced adherence with medication. The paper will further discuss the role of psychologic therapy in improving treatment adherence in children and adolescents with ALL, with a particular focus on identifying the need for controlled outcome studies.

Thyroid neoplasia following irradiation in adolescent and young adult survivors of childhood cancer.

OBJECTIVES: To describe a cohort of survivors of childhood malignancy at risk of developing thyroid abnormality, and propose guidelines for management of such patients. DESIGN: Retrospective case series. SETTING: Late-effects oncology clinic at a large children's hospital in Sydney. SUBJECTS: 142 patients who had received irradiation to the thyroid from the 1970s onwards, who attended the late-effects clinic from May 1989 to December 1998. INTERVENTIONS: Thyroid palpation by an endocrinologist or surgeon, serum thyroid-stimulating hormone assay and thyroid ultrasound examination were performed on all subjects and, depending on findings, some subjects proceeded to fine-needle biopsy or surgery (total thyroidectomy). A few patients required adjuvant (131)I administration. OUTCOME MEASURES: Radiation dose received; results of thyroid palpation; thyroid function tests; ultrasound findings; diagnosis of the abnormalities; and outcomes of surgical interventions. RESULTS: 49 subjects (24 of 65 patients who received scatter irradiation to the thyroid and 25 of 78 patients who received direct irradiation) had thyroid surgery. Of these, 12 in the scatter and six in the direct irradiation group were found to have thyroid malignancy. Fifty subjects with abnormal ultrasound results remain under surveillance. Having a palpable thyroid was predictive of malignancy, but age at original diagnosis, sex, current age, time since irradiation, radiation dose, nodule type and nodal involvement were not. CONCLUSION: There is a significant risk of cancer in thyroid glands exposed to radiation as part of therapy for childhood cancer. This risk is greater for patients who received scatter (versus direct) irradiation. Nodular change is usually not apparent for many years, so lifelong surveillance is necessary. Palpation alone is not sufficient to detect thyroid cancer and thyroid ultrasound examination is recommended.