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Early human intervention

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

Huiyuan Li, Yuan Xu, Yunfei Chen, Lulu Ji, Yanmei Xu, Wenting Zheng, Ting Sun, Rongfeng Fu, Xiaolei Pei, Xiaofan Liu, Feng Xue, Wei Liu, Wentian Wang, Ying Chi, Renchi Yang, Jun Wei, Lei Zhang

Nature Medicine · 2026 · 32(6):2026–2036

Original publication PubMed

Study at a glance

Design
Single-arm, open-label phase 1/2 clinical trial with mechanistic mouse and cellular experiments
Population or model
Adults with steroid-refractory or steroid-dependent immune thrombocytopenia
Sample size
25 enrolled patients
Intervention
Oral NMN 450 mg twice daily for 2 weeks
Publication type
Phase 1/2 single-arm clinical trial
Evidence class
human-study
Research area
Immune thrombocytopenia and immunometabolism
Trial registration
NCT06776510

Study endpoints

  • Safety and tolerability
  • Primary platelet response within 2 weeks
  • Platelet-count kinetics through follow-up
  • Immunoglobulin levels and infections
  • Mechanistic macrophage and NAD-related measures

Plain-language summary

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.

Limitations

  • 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.

Funding and transparency

Funding

Supported by the National Natural Science Foundation of China (82570173 to H.L., 82470136 to R.Y., and 82341214 to J.W.); National Key Research and Development Program of China (2023YFC2507802 to F.X.); CAMS Innovation Fund for Medical Sciences (2024-I2M-ZH-016 and 2025-I2M-XHZY-034 to L.Z.); Tianjin Municipal Science and Technology Commission (25ZXZSSS00510 to L.Z.); and Distinguished Young Scholars of Tianjin (22JCJQJC00070 to J.W.).

Competing interests

The authors declare no competing interests.

HealthspanX observation

This is disease-treatment research in a refractory ITP population, not consumer longevity evidence.

Interpretive weight

Limited

Prospective phase 1/2 human study in a specific refractory autoimmune disease, but single-arm, open-label and only 25 patients.

This record summarizes a publication; it is not medical advice or a claim that a HealthspanX product reproduces the study's results. Report a correction.

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