Purpose Global data mapping access to essential chemotherapeutics for pediatric cancer are scarce. We report a survey of international pediatric cancer care providers’ access to these medicines. Methods A Web-based survey was sent to pediatric oncologists registered on the Cure4Kids Web portal. We queried chemotherapeutics in the WHO Essential Medicines List for Children, from which the average proportional availability was summarized as each country’s access score. In addition, we examined availability of drug packages defined by the WHO-sanctioned Expert Committee for eight pediatric cancers. We undertook a sensitivity analysis investigating how regimen access would change if the cytotoxics specified in recent agreements between the Clinton Health Access Initiative, American Cancer Society, and pharmaceutical companies were universally available. Results There were significant ( P < .001) differences in the median access scores between World Bank income groups, and 42.9% of respondents from low-income and lower middle–income countries reported suboptimal access scores. Our disease-based analysis revealed that 42.1% of patients in low-income and lower middle–income countries lacked full access to chemotherapy packages. Guaranteed availability of the cytotoxics specified in the Clinton Health Access Initiative/American Cancer Society agreements was projected to increase this regimen-based access by 1.6%, although including four additional chemotherapeutics would further increase coverage by 13.9%. Conclusion This study is the first, to our knowledge, to assess worldwide variation in practical access to pediatric chemotherapy. Although mapping the proportion of available chemotherapeutics is informative, we also developed a meaningful estimate of access using disease-specific drug packages. These data provide an important baseline for continued monitoring and can aid in planning adaptive treatment guidelines that consider the trade-offs between access and outcomes.
GM-CSF, in addition to IS, may shorten time to neutrophil count recovery, may be beneficial in decreasing infection rates, and may improve platelet response in patients with SAA. However, consistent with studies utilizing G-CSF, GM-CSF probably does not affect overall response rate. To fully answer whether or not cytokine therapy is of added value to IS in pediatric patients, a multi-institutional randomized trial is needed.
Hepatic sequestration is an uncommon complication in patients with homozygous sickle cell disease. Although transfusion therapy has been effective for the acute condition, no definitive treatment of chronic hepatic sequestration has been identified. We describe a 17-year-old male patient with hemoglobin SS and chronic hepatic sequestration who was treated with long-term (60 months) hydroxyurea. After 36 months of HU therapy, the patient had both an excellent hematologic response and a resolution of hepatic sequestration, as evidenced by disappearance of clinical hepatomegaly, normalization of liver volume on serial computed tomography scans, as well as decreased sinusoidal dilatation and congestion and red blood cell sickling on liver biopsy. The findings in this case suggest that hydroxyurea may benefit patients who have unusual complications of sickle cell disease, such as chronic erythrocyte sickling in the liver.
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