Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial
Huiyuan Li et al. · Nature Medicine · 2026
Evidence type: Early human interventionInterpretive weight: LimitedResearch area: Immune thrombocytopenia and immunometabolism
DesignSingle-arm, open-label phase 1/2 clinical trial with mechanistic mouse and cellular experiments
PopulationAdults with steroid-refractory or steroid-dependent immune thrombocytopenia
Sample25 enrolled patients
InterventionOral NMN 450 mg twice daily for 2 weeks
EndpointsSafety and tolerability; Primary platelet response within 2 weeks; Platelet-count kinetics through follow-up; Immunoglobulin levels and infections; Mechanistic macrophage and NAD-related measures
What the publication reported
This Nature Medicine study combined extensive mechanistic experiments with an open-label phase 1/2 trial in 25 adults with steroid-refractory or steroid-dependent immune thrombocytopenia.
Participants received 450 mg NMN twice daily for two weeks. No dose-limiting toxicities or treatment-related serious adverse events were reported. Five patients, 20%, met the prespecified primary platelet-response endpoint.
Exploratory analyses reported broader platelet increases in some participants, but there was no randomized control group. The result is disease-specific early-phase evidence and cannot be generalized to routine supplementation or longevity.
Key findings
No dose-limiting toxicities or treatment-related serious adverse events were reported.
Mild treatment-related adverse events occurred in 12% of participants.
Five of 25 patients met the prespecified primary platelet-response endpoint.
Exploratory analyses found that 60% exceeded 1.5 times baseline platelet count during treatment and 52% maintained responses through week 8.
Mechanistic experiments linked CD38-NAD biology with macrophage polarization and platelet clearance.
What it cannot establish
Single-arm, open-label design without placebo control.
Only 25 patients.
Short two-week treatment period.
Highly specific refractory autoimmune-disease population.
Mechanistic mouse and cell findings do not establish general supplementation benefits.
Further controlled trials are needed to determine efficacy.
HealthspanX claim boundary: This study does not establish NMN as an approved treatment for ITP or other autoimmune disease and does not support general health, anti-aging or longevity claims.
