Simple Interventions Can Improve Access to Autologous Stem Cell Transplant in Low- and Lower-Middle-Income Countries by Reducing Complexity, Lowering Costs, and Minimizing Infectious Risks.

BACKGROUND

The aim was to enhance access to autologous stem cell transplant (ASCT) for high-risk neuroblastoma in a public hospital in a lower-middle-income country, while ensuring safety.

METHODS

Data from a single center were analyzed in this ambispective, observational study spanning 12 years (2012-2023). The adaptations included performing ASCT in isolation rooms within the general ward, storing stem cells at 2°C-6°C without cryopreservation for 7 days, utilizing plerixafor for stem cell mobilization, no central lines, minimizing venipunctures for sampling, no antibacterial/antifungal/antiviral prophylaxis, administering pegylated-G-CSF on Day +4, avoiding platelet transfusion for asymptomatic thrombocytopenia, prednisolone for preventing engraftment syndrome, and early discharge upon rising white cell count and monocytosis.

RESULTS

Seventy-three patients were treated on the backbone of the high-risk neuroblastoma study-1 of SIOP-Europe (SIOPEN). The median age was 38 months. Sixty-six (90.4%) patients had Stage 4 disease. A regimen of busulfan+melphalan was administered to 62 (84.9%) patients. Median CD34 cell count and stem cell viability were 5.2 × 106/kg and 91.5%. The median hospital stay was 20 days. At discharge, the median absolute neutrophil and platelet counts were 0.60 × 109/L and 19 × 109/L. After 2017, there was a reduction in the number of aphereses (p < 0.001), blood transfusions (p = 0.003), amphotericin (p = 0.012), and venipunctures (p = 0.045). Forty (54.8%) patients died; relapse: 33 (82.5%); progressive disease: five (12.5%); and treatment-related mortality: two (5%). There was no case of engraftment failure. The median follow-up for surviving patients was 55.3 months. Five-year overall survival (OS) and event-free survival (EFS) were 34.6% ± 6.6% and 27% ± 5.8%. Survival was comparable to our historical cohort of 28 patients treated without ASCT (4-year EFS: 29.3%; 4-year OS: 29.3%).

CONCLUSION

In our cohort, the incorporation of ASCT into multimodality therapy was feasible with resource-adapted strategies; however, this did not translate into a meaningful improvement in survival outcomes when compared with our historical non-transplant cohort.