Tier 3 — preclinical

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

Bibliography

PubMed
PMID 42056497
DOI
10.1038/s41591-026-04366-x
Funding
Supported by National Natural Science Foundation of China grants 82570173 (Huiyuan Li), 82470136 (Renchi Yang) and 82341214 (Jun Wei); National Key Research and Development Program of China grant 2023YFC2507802 (Feng Xue); CAMS Innovation Fund for Medical Sciences grants 2024-I2M-ZH-016 and 2025-I2M-XHZY-034 (Lei Zhang); Tianjin Municipal Science and Technology Commission grant 25ZXZSSS00510 (Lei Zhang); and Distinguished Young Scholars of Tianjin grant 22JCJQJC00070 (Jun Wei).
Competing interests
The authors declare no competing interests.

Study snapshot

DesignSingle-arm, open-label phase 1/2 clinical trial.
ModelAdults with steroid-refractory or steroid-dependent immune thrombocytopenia.
Samplen=25.
InterventionOral NMN 450 mg twice daily (900 mg/day).
Duration2 weeks of NMN treatment with follow-up through week 8.
EndpointsSafety and tolerability; Platelet response ≥50 × 10^9/L within 2 weeks; Platelet count relative to baseline; Durability through week 8; Immune and CD38–NAD+ mechanistic measures

What the study showed, in plain terms

This phase 1/2 study moved NMN beyond healthy volunteers into immune thrombocytopenia. Twenty-five adults with steroid-refractory or steroid-dependent ITP received 900 mg/day NMN for two weeks without a placebo group.

No dose-limiting toxicity or treatment-related serious adverse event was reported. Five of 25 participants met the prespecified platelet-response endpoint, while exploratory analyses found larger platelet increases in additional participants and persistence in some through week eight. Because the trial was single-arm, efficacy must be confirmed in a randomized study.

Key findings

  • No dose-limiting toxicities or treatment-related serious adverse events were reported.
  • Mild treatment-related adverse events occurred in about 12% of participants.
  • Five of 25 participants (20%) met the primary platelet-response endpoint within two weeks.
  • Exploratory analyses reported platelet counts above 1.5× baseline in 60% and maintained responses through week 8 in 52%.
  • The study linked the clinical intervention to CD38–NAD+ immunometabolic biology.

What this study can and cannot tell us

This was a 25-person single-arm early-phase study, so spontaneous variation, concomitant treatment and regression to the mean cannot be excluded. The response data are promising but not proof that NMN is an effective ITP therapy.

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Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology