A Case Report of Selinexor in the Treatment of Post-Transplant Relapsed Acute Myeloid Leukemia with Minimal Differentiation (AML-M0)
Year: 2026
Type: article
Abstract: Acute myeloid leukemia with minimal differentiation (AML-M0) is a clinically rare high-risk subtype of acute myeloid leukemia. Tumor cells of this subtype have an extremely low degree of differentiation, making morphological identification challenging. It is characterized by high overall malignancy, frequent primary drug resistance and a high recurrence rate, leading to extremely poor long-term prognosis in pediatric patients. Currently, there is no unified standard salvage treatment regimen for this condition. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) serves as a critical curative approach for high-risk acute myeloid leukemia. Nevertheless, some pediatric patients still experience disease relapse after multi-line chemotherapy and initial transplantation. Conventional salvage chemotherapy yields a low re-remission rate and short remission duration, which remains a major focus and challenge in clinical management. Selinexor, a novel oral selective inhibitor of exportin 1 (XPO1), exerts anti-leukemic effects by reversing tumor drug resistance and promoting apoptosis of leukemic cells, showing promising application prospects in relapsed/refractory acute myeloid leukemia. This article reports an 8-year-old pediatric patient with AML-M0, who suffered multiple relapses following conventional multi-regimen chemotherapy and the first haploidentical donor (father) allo-HSCT, and developed resistance to multi-line salvage chemotherapy. Salvage therapy with selinexor combined with chemotherapy was administered at our center as a desperation measure in this ultra-high-risk setting after all standard curative options had been exhausted. The patient achieved deep complete remission again and was successfully bridged to a second allo-HSCT; as of the last follow-up, the patient remained in continuous complete remission, although the observation period after the second (April 2026) transplantation is still limited. Given the absence of prospective safety data for this specific four-drug combination—particularly in a child with pre-existing pulmonary chronic GVHD and impaired ventilatory function—potential uncharacterized synergistic toxicities must be emphasized, and this experience should be interpreted strictly as a single-case desperation measure that cannot be recommended or generalized without formal clinical trials. Combined with the diagnosis and treatment course of this case and relevant domestic and international literature, this paper discusses the potential clinical efficacy, medication safety and application value of selinexor in pediatric patients with relapsed/refractory acute myeloid leukemia after transplantation. We emphasize that this four-drug regimen constituted a desperation measure in an ultra-high-risk setting with no remaining standard curative options, that it carries unknown synergistic toxicities, and that it cannot be recommended or generalized without formal prospective clinical trials, although it may provide a reference for individualized treatment of comparable high-risk pediatric patients after multidisciplinary evaluation.
Source: Transplantology
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $1,207
OpenAlex ID: W7225647867