RESUMO
Annually, over 400,000 children develop cancer, with the majority living in low- and middle-income countries (LMICs). Survival rates in high-income countries (HICs; ≥ 75%-80%) significantly exceed those in LMICs (< 30%). Acute myeloid leukemia (AML) is a childhood cancer with high mortality rates in LMICs and is not included in the World Health Organization (WHO)'s 'six common and curable types of cancer'. This case report explores two pediatric AML cases in Kenya (LMIC) and the Netherlands (HIC), highlighting differences and similarities in both patient journeys. The first case is a 15-year-old Kenyan boy who initially experienced dizziness and fatigue. After repeated blood transfusions without a definitive diagnosis, AML was confirmed via bone marrow aspiration (BMA) 63 days later, and treatment followed the SIOP PODC AML guidelines for LMICs. The second case is a 6-year-old Dutch boy with fatigue and malaise. Initially diagnosed with post-viral bone marrow failure, a BMA performed 61 days after symptom onset revealed AML, and treatment followed the NOPHO-DBH AML-2012 protocol. Both patients faced frequent febrile neutropenia, managed per local guidelines, illustrating the balance between anti-cancer treatment and supportive care. Despite challenges, both boys completed treatment and are in complete remission. This case series highlights the potential for effective AML treatment in resource-constrained settings and underscores the need to address cancers beyond the 'six common and curable types'.
RESUMO
BACKGROUND: Alternative treatments for visceral leishmaniasis (VL) are required in East Africa. Paromomycin sulphate (PM) has been shown to be efficacious for VL treatment in India. METHODS: A multi-centre randomized-controlled trial (RCT) to compare efficacy and safety of PM (20 mg/kg/day for 21 days) and PM plus sodium stibogluconate (SSG) combination (PM, 15 mg/kg/day and SSG, 20 mg/kg/day for 17 days) with SSG (20 mg/kg/day for 30 days) for treatment of VL in East Africa. Patients aged 4-60 years with parasitologically confirmed VL were enrolled, excluding patients with contraindications. Primary and secondary efficacy outcomes were parasite clearance at 6-months follow-up and end of treatment, respectively. Safety was assessed mainly using adverse event (AE) data. FINDINGS: The PM versus SSG comparison enrolled 205 patients per arm with primary efficacy data available for 198 and 200 patients respectively. The SSG & PM versus SSG comparison enrolled 381 and 386 patients per arm respectively, with primary efficacy data available for 359 patients per arm. In Intention-to-Treat complete-case analyses, the efficacy of PM was significantly lower than SSG (84.3% versus 94.1%, differenceâ=â9.7%, 95% confidence interval, CI: 3.6 to 15.7%, pâ=â0.002). The efficacy of SSG & PM was comparable to SSG (91.4% versus 93.9%, differenceâ=â2.5%, 95% CI: -1.3 to 6.3%, pâ=â0.198). End of treatment efficacy results were very similar. There were no apparent differences in the safety profile of the three treatment regimens. CONCLUSION: The 17 day SSG & PM combination treatment had a good safety profile and was similar in efficacy to the standard 30 day SSG treatment, suggesting suitability for VL treatment in East Africa. CLINICAL TRIALS REGISTRATION: www.clinicaltrials.govNCT00255567.