Patients undergoing hematopoietic stem cell transplantation (HCT) are expected to have severe thrombocytopenia. We have been through several blood shortages e.g. holidays, viral outbreaks, live mass vaccinations, environmental disasters and lack of public education to increase volunteer donations. Some patients refuse transfusions due to religious convictions, such as the Jehovah’s Witnesses. In the latter, hematologic support may be limited, potentially compromising the safety of the procedure. Romiplostim, a thrombopoietin receptor agonist, has shown promise in refractory thrombocytopenia scenarios, but its use in the HCT setting remains underexplored.

Our objective was to investigate the role and safety of romiplostim as an adjuvant agent for platelet support in pediatric HCT to reduce platelet transfusion requirement, prevent adverse effects and alloimmunization, and also include patients with transfusion objection based on religious beliefs.

Romiplostim was administered at 5 µg/kg/week subcutaneous starting on day +2, and continued until hematologic recovery.

Our first patient was a 6 year-old girl diagnosed with stage IV neuroblastoma, previously treated with multiple therapeutic lines and subsequently referred for an HLA-identical related myeloablative HCT with busulfan-melphalan, with restriction to blood transfusions due to religious reasons. With the use of Romiplostim she maintained platelet counts above the critical levels of 10,000/mm³ for most of the HCT course. She received only one platelet transfusion on day +10 due to a lower platelet count, fever and sepsis, from which she completely recovered. Neutrophil engraftment was achieved on day +10 and platelet engraftment on day +17. No severe hemorrhagic events or romiplostim-related toxicities were observed.

Romiplostim proved to be a safe and effective alternative for managing thrombocytopenia in this first pediatric HCT patient with transfusion restrictions. This experience broadens the potential use of the agent, suggesting its applicability as a personalized therapeutic strategy in the HCT setting. Its use may be considered not only in cases of religious objection, but also as an approach to minimize transfusions and prevent alloimmunization. Further studies are needed to validate its efficacy on a larger scale, define cost-efficiency and support its integration into clinical practice.