A case report published recently in Frontiers in Immunology describes the successful treatment of a 6-year-old boy with systemic mastocytosis with associated hematologic neoplasm (SM-AHN) using the targeted therapy drug gemtuzumab ozogamicin.
The young child was diagnosed with SM with AML1::ETO-positive acute myeloid leukemia (AML), a rare type of blood cancer caused by a genetic mutation that leads to the growth of abnormal mast cells in the body. Prognosis for this condition tends to be poor, with limited therapeutic options.
After the boy showed no response to conventional treatment (including three rounds of chemotherapy), researchers treated him with gemtuzumab ozogamicin, an antibody drug which targets cells that carry a protein called CD33. They decided on this therapy because the boy had a high expression of CD33 on both leukemic cells and abnormal mast cells throughout his body, the study’s authors reported.
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After two treatment cycles of gemtuzumab ozogamicin, the child experienced rapid remission. The authors note another case report in the medical literature where gemtuzumab ozogamicin resulted in the sustained remission in a patient with refractory mast cell leukemia.
The study’s authors say that although these are single cases and more research is needed to validate the findings, the results warrant further investigation into the efficacy and safety of gemtuzumab ozogamicin in more patients with similar diagnoses.
“Our successful case study, coupled with pertinent literature, has facilitated the progression of future clinical trials involving GO [gemtuzumab ozogamicin] for the treatment of SM-AHN,” they write. “Despite inherent limitations that may compromise the robustness of its conclusions, this case report still warrants recommending the therapeutic approach. The aim is to explore its potential benefits in patients with a poor prognosis.”
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