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, Sun T, Fu R, Pei X, Liu X, Xue F, Liu W and 5 more

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Wang W, Chi Y, Yang R, Wei J, Zhang L

Nature medicine29 Apr 2026Cited by 3 (Europe PMC)

PMID42056497DOI10.1038/s41591-026-04366-xPepBankPUB-42056497

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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study13

13 results from studies that gave the peptide. 8 results from people.

01HumanNon-randomized trialSafety outcome
In adults with steroid-refractory or steroid-dependent ITP receiving NMN, no dose-limiting toxicities occurred.
In plain wordsIn adults with difficult-to-treat ITP receiving NMN, no dose-limiting toxicities occurred.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicableDose-limiting toxicity
Study
n = 25
Analysis
Primary
Outcome group
Adverse events · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Source sentences
Summary
Among 25 enrolled patients, no dose-limiting toxicities or treatment-related serious adverse events occurred;
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649706
02HumanNon-randomized trialSafety outcome
In adults with ITP, no treatment-related serious adverse events occurred with NMN.
In plain wordsIn adults with ITP, no serious adverse events related to NMN occurred.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicableTreatment-related serious adverse events
Study
n = 25
Analysis
Primary
Outcome group
Adverse events · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Source sentences
Summary
Among 25 enrolled patients, no dose-limiting toxicities or treatment-related serious adverse events occurred;
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649707
03HumanNon-randomized trialSafety outcome
In adults with ITP, mild treatment-related adverse events occurred in 12% with NMN.
In plain wordsIn adults with ITP, 12% had mild adverse events related to NMN.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicableMild treatment-related adverse events
Study
n = 25
Analysis
Primary
Outcome group
Adverse events · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Measurements
Mild treatment-related adverse events
12%
NMN-treated patients
Source sentences
Summary
NMN was well tolerated, with only mild treatment-related adverse events in 12% and non-severe infections (grade 1) in 8% of patients
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649708
04HumanNon-randomized trialSafety outcome
In adults with ITP receiving NMN, grade 1 infections occurred in 8%.
In plain wordsIn adults with ITP receiving NMN, 8% had non-severe infections.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicableNon-severe infections
Study
n = 25
Analysis
Primary
Outcome group
Adverse events · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Measurements
Non-severe infections
8%
NMN-treated patients
Source sentences
Summary
NMN was well tolerated, with only mild treatment-related adverse events in 12% and non-severe infections (grade 1) in 8% of patients
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649709
05HumanNon-randomized trialEfficacy outcome
In adults with ITP receiving NMN, five patients (20.0%) met the primary platelet-response endpoint.
In plain wordsIn adults with ITP receiving NMN, five patients (20.0%) met the primary platelet-response endpoint.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicablePrimary platelet response
Study
n = 25
Analysis
Primary
Outcome group
Blood counts and hematopoiesis · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Measurements
Platelet response rate
20%
NMN
Source sentences
Summary
Five patients (20.0%) met the primary platelet-response endpoint.
2 context sentences(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Summary · context
Primary endpoints were safety/tolerability and platelet response (≥50 × 10⁹ per liter within 2 weeks, confirmed by two consecutive measurements one or more days apart, without rescue therapy or dose escalation of thrombopoietin receptor agonists or corticosteroids).
Report a problemFND-4205649711
06HumanNon-randomized trialEfficacy outcome
In adults with ITP receiving NMN, 60% achieved platelet counts above 1.5-fold baseline during treatment.
In plain wordsIn adults with ITP receiving NMN, 60% reached platelet counts above 1.5 times their starting level.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
IncreasePlatelets above 1.5-fold baseline
Study
n = 25
Analysis
Exploratory
Outcome group
Blood counts and hematopoiesis · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Measurements
Platelet count responders
60%
More than 1.5-fold baseline during treatment
Source sentences
Summary
In exploratory analyses, overall, 60% of patients achieved platelet counts more than 1.5× baseline during treatment, and 52% maintained responses through week 8.
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649712
07HumanNon-randomized trialEfficacy outcome
In adults with ITP receiving NMN, 52% maintained responses through week 8.
In plain wordsIn adults with ITP receiving NMN, 52% maintained responses through week 8.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Not applicableResponse maintained through week 8
Study
n = 25
Analysis
Exploratory
Outcome group
Blood counts and hematopoiesis · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Measurements
Maintained response
52%
Through week 8
Source sentences
Summary
In exploratory analyses, overall, 60% of patients achieved platelet counts more than 1.5× baseline during treatment, and 52% maintained responses through week 8.
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649713
08HumanNon-randomized trialSafety outcome
In adults with ITP receiving NMN, immunoglobulin levels remained stable.
In plain wordsIn adults with ITP receiving NMN, antibody levels remained stable.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
No significant changeImmunoglobulin levels
Study
n = 25
Outcome group
Immune response · Immune and inflammation
Studied dose
450 mg twice daily · oral · 2 weeksAs given in this study (human).
Source sentences
Summary
while immunoglobulin levels remained stable, consistent with preserved humoral immunity.
1 context sentence(design, population, supporting numbers)
Summary · context
Based on these findings, we conducted a single-arm, open-label phase 1/2 trial of low-dose oral NMN (450 mg twice daily for 2 weeks) in adults with steroid-refractory or steroid-dependent ITP.
Report a problemFND-4205649710
09AnimalAnimal study (in vivo)Mechanism
In mice, NMN supplementation restored NAD levels.
In plain wordsIn mice, NMN restored NAD levels.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
IncreaseNAD restoration
Study
mousen: not reported in source
Thrombocytopenia model
Outcome group
Receptor activity and mechanism · Immune and inflammation
Studied dose
Dose not stated
Source sentences
Summary
In mice, CD38 inhibition or nicotinamide mononucleotide (NMN) supplementation restores NAD⁺, reprograms macrophages, downregulates FcγRI and prevents thrombocytopenia.
Report a problemFND-4205649701
10AnimalAnimal study (in vivo)Mechanism
In mice, NMN supplementation reprogrammed macrophages.
In plain wordsIn mice, NMN changed macrophage programming.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
Direction not reportedMacrophage reprogramming
Study
mousen: not reported in source
Thrombocytopenia model
Outcome group
Immune response · Immune and inflammation
Studied dose
Dose not stated
Source sentences
Summary
In mice, CD38 inhibition or nicotinamide mononucleotide (NMN) supplementation restores NAD⁺, reprograms macrophages, downregulates FcγRI and prevents thrombocytopenia.
Report a problemFND-4205649702
11AnimalAnimal study (in vivo)Mechanism
In mice, NMN supplementation downregulated FcγRI.
In plain wordsIn mice, NMN reduced FcγRI expression.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
DecreaseFcγRI expression
Study
mousen: not reported in source
Thrombocytopenia model
Outcome group
Immune response · Immune and inflammation
Studied dose
Dose not stated
Source sentences
Summary
In mice, CD38 inhibition or nicotinamide mononucleotide (NMN) supplementation restores NAD⁺, reprograms macrophages, downregulates FcγRI and prevents thrombocytopenia.
Report a problemFND-4205649703
12AnimalAnimal study (in vivo)Efficacy outcome
In mice, NMN supplementation prevented thrombocytopenia.
In plain wordsIn mice, NMN prevented low platelet counts.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
DecreaseThrombocytopenia
Study
mousen: not reported in source
Thrombocytopenia model
Outcome group
Blood counts and hematopoiesis · Immune and inflammation
Studied dose
Dose not stated
Source sentences
Summary
In mice, CD38 inhibition or nicotinamide mononucleotide (NMN) supplementation restores NAD⁺, reprograms macrophages, downregulates FcγRI and prevents thrombocytopenia.
Report a problemFND-4205649704
13AnimalAnimal study (in vivo)Safety outcome
In an ovalbumin immunization model, NMN did not impair antigen-specific antibody production.
In plain wordsIn an animal immunization model, NMN did not impair production of antibodies against the antigen.
Peptide
NMN (nicotinamide mononucleotide)
Outcome
No significant changeAntigen-specific antibody production
Study
n: not reported in source
Ovalbumin immunization model
Outcome group
Immune response · Immune and inflammation
Studied dose
Dose not stated
Source sentences
Summary
In an ovalbumin immunization model, NMN treatment does not impair antigen-specific antibody production, supporting preservation of humoral responses.
Report a problemFND-4205649705

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 ongoing · source ClinicalTrials.gov (opens in a new tab)

RecruitingPhase 1/2NCT06776510

A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia

Institute of Hematology & Blood Diseases Hospital, Chinan = 2015 Jan 2025 to est. 1 Mar 2026

Immune Thrombocytopenia · Treatment

Trial page ClinicalTrials.govUpdated 5 Aug 2025

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
21 Oct 2026, 15:58 UTC
Study results updated
Version 1
21 Oct 2026, 12:50 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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MeSH terms

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