Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial

Li H, Xu Y, Chen Y, Ji L, Xu Y, Zheng W · Nature Medicine

Five of 25 patients (20%) met the primary platelet-response endpoint; 60% reached platelet counts above 1.5 times baseline during treatment and 52% held a response to week 8. No dose-limiting toxicities or treatment-related serious adverse events; mild treatment-related events in 12%. The rationale is the CD38-NAD+ axis: restoring macrophage NAD+ downregulates FcγRI and reduces platelet phagocytosis.

Population

Adults with steroid-refractory or steroid-dependent immune thrombocytopenia

Intervention

Oral NMN 450 mg twice daily for 2 weeks

Comparator

None — single-arm, open-label

Limitations

Single-arm and open-label — no placebo comparison for a condition with spontaneous fluctuation. Twenty-five patients, two weeks of dosing. A disease indication, not a healthspan endpoint.