Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (611 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
A humoral factor that stimulates the production of thrombocytes (blood platelets). Thrombopoietin stimulates the proliferation of bone marrow megakaryocytes and their release of blood platelets. The process is called thrombopoiesis.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Thrombocytopoiesis-Stimulating Factor; Thrombocytopoiesis Stimulating Factor; MGDF Factor; mpl Ligand; Ligand, mpl; c-mpl Ligand; Ligand, c-mpl; Megakaryocyte Growth and Development Factor.
Where it fits in the MeSH classification
- Broader topics
- Colony-Stimulating Factors
Related concepts
- Blood Platelets
- Megakaryocytes
- Thrombopoiesis
Research landscape
PubMed indexes 3,604 records tagged with the MeSH heading “Thrombopoietin” (counts retrieved 2026-10-31). 538 of them (14.9%) were published from 2020 onward, and the busiest period so far is 2000-2009 with 1,156 records.
Reviews account for 621 records (17.2%) and meta-analyses for 31. Randomized controlled trials number 95 (2.6%) as a share of all records on the topic.
Show the data
| Period | Records |
|---|---|
| through 1999 | 965 |
| 2000-2009 | 1,156 |
| 2010-2019 | 945 |
| 2020 onward | 538 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 621 | 17.2% |
| Meta-analyses | 31 | 0.9% |
| Randomized controlled trials | 95 | 2.6% |
Method: PubMed query "Thrombopoietin"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-31.
Reading the evidence in drugs and chemicals
Studies of a chemical or drug can answer very different questions: what it is and how it behaves in cells and animals, how the body handles it, and whether it changes outcomes in people. Laboratory and animal findings often do not carry over to patients, and observational studies of exposures are confounded by who uses a substance and why. Nothing on this page is a recommendation to use, avoid, start or stop any substance or medicine; talk to a qualified clinician or pharmacist about your own situation. Check the dose, the species, the comparison group and whether a patient-relevant outcome was measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Cytokine-induced megakaryocytic differentiation is regulated by genome-wide loss of a uSTAT transcriptional program | 2016 | Research article Design not recorded in structured metadata | 67 | cc by PMID 26702099 · DOI · PMC4801948 |
| STAT5-regulated microRNA-193b controls haematopoietic stem and progenitor cell expansion by modulating cytokine receptor signalling | 2015 | Research article Design not recorded in structured metadata | 46 | cc by PMID 26603207 · DOI · PMC4674773 |
| Thrombopoietin mutation in congenital amegakaryocytic thrombocytopenia treatable with romiplostim | 2018 | Research article Design not recorded in structured metadata | 46 | cc by PMID 29191945 · DOI · PMC5760853 |
| Romiplostim-N01 for cancer therapy-induced thrombocytopenia: A propensity score-matched cohort study | 2026 | Research article Design not recorded in structured metadata | 1 | cc by PMID 42216387 · DOI · PMC13225505 |
Cytokine-induced megakaryocytic differentiation is regulated by genome-wide loss of a uSTAT transcriptional program [1]
Abstract reading summary — source excerpts
- Opening excerpt
- Metazoan development is regulated by transcriptional networks, which must respond to extracellular cues including cytokines.
- Closing excerpt
- These results identify a previously unrecognized mechanism of cytokine-mediated differentiation.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (144 words)
Metazoan development is regulated by transcriptional networks, which must respond to extracellular cues including cytokines. The JAK/STAT pathway is a highly conserved regulatory module, activated by many cytokines, in which tyrosine-phosphorylated STATs (pSTATs) function as transcription factors. However, the mechanisms by which STAT activation modulates lineage-affiliated transcriptional programs are unclear. We demonstrate that in the absence of thrombopoietin (TPO), tyrosine-unphosphorylated STAT5 (uSTAT5) is present in the nucleus where it colocalizes with CTCF and represses a megakaryocytic transcriptional program. TPO-mediated phosphorylation of STAT5 triggers its genome-wide relocation to STAT consensus sites with two distinct transcriptional consequences, loss of a uSTAT5 program that restrains megakaryocytic differentiation and activation of a canonical pSTAT5-driven program which includes regulators of apoptosis and proliferation. Transcriptional repression by uSTAT5 reflects restricted access of the megakaryocytic transcription factor ERG to target genes. These results identify a previously unrecognized mechanism of cytokine-mediated differentiation.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 26702099 · DOI · PMC4801948
STAT5-regulated microRNA-193b controls haematopoietic stem and progenitor cell expansion by modulating cytokine receptor signalling [2]
Abstract reading summary — source excerpts
- Opening excerpt
- Haematopoietic stem cells (HSCs) require the right composition of microRNAs (miR) for proper life-long balanced blood regeneration.
- Closing excerpt
- This STAT5-induced microRNA provides a negative feedback for excessive signalling to restrict uncontrolled HSC expansion.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (149 words)
Haematopoietic stem cells (HSCs) require the right composition of microRNAs (miR) for proper life-long balanced blood regeneration. Here we show a regulatory circuit that prevents excessive HSC self-renewal by upregulation of miR-193b upon self-renewal promoting thrombopoietin (TPO)-MPL-STAT5 signalling. In turn, miR-193b restricts cytokine signalling, by targeting the receptor tyrosine kinase c-KIT. We generated a miR-193b knockout mouse model to unravel the physiological function of miR-193b in haematopoiesis. MiR-193b(-/-) mice show a selective gradual enrichment of functional HSCs, which are fully competent in multilineage blood reconstitution upon transplantation. The absence of miR-193b causes an accelerated expansion of HSCs, without altering cell cycle or survival, but by decelerating differentiation. Conversely, ectopic miR-193b expression restricts long-term repopulating HSC expansion and blood reconstitution. MiR-193b-deficient haematopoietic stem and progenitor cells exhibit increased basal and cytokine-induced STAT5 and AKT signalling. This STAT5-induced microRNA provides a negative feedback for excessive signalling to restrict uncontrolled HSC expansion.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 26603207 · DOI · PMC4674773
Thrombopoietin mutation in congenital amegakaryocytic thrombocytopenia treatable with romiplostim [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited disorder characterized at birth by thrombocytopenia with reduced megakaryocytes, which evolves into generalized bone marrow aplasia during childhood.
- Closing excerpt
- Recognizing patients with THPO mutations among those with juvenile bone marrow failure is essential to provide them with appropriate substitutive therapy and prevent the use of invasive and unnecessary treatments, such as hematopoietic stem cell transplantation or immunosuppression.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (171 words)
Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited disorder characterized at birth by thrombocytopenia with reduced megakaryocytes, which evolves into generalized bone marrow aplasia during childhood. Although CAMT is genetically heterogeneous, mutations of MPL, the gene encoding for the receptor of thrombopoietin (THPO), are the only known disease-causing alterations. We identified a family with three children affected with CAMT caused by a homozygous mutation (p.R119C) of the THPO gene. Functional studies showed that p.R119C affects not only ability of the cytokine to stimulate MPL but also its release, which is consistent with the relatively low serum THPO levels measured in patients. In all the three affected children, treatment with the THPO-mimetic romiplostim induced trilineage hematological responses, remission of bleeding and infections, and transfusion independence, which were maintained after up to 6.5 years of observation. Recognizing patients with THPO mutations among those with juvenile bone marrow failure is essential to provide them with appropriate substitutive therapy and prevent the use of invasive and unnecessary treatments, such as hematopoietic stem cell transplantation or immunosuppression.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 29191945 · DOI · PMC5760853
Romiplostim-N01 for cancer therapy-induced thrombocytopenia: A propensity score-matched cohort study [4]
Abstract reading summary — source excerpts
- Opening excerpt
- Cancer therapy-induced thrombocytopenia (CTIT) is a common complication in patients with solid tumors during their cancer treatment.
- Closing excerpt
- These findings position romiplostim-N01 as a favorable alternative to rhIL-11 or rhTPO in CTIT, given its enhanced early PLT response and reduced risk of chemotherapy disruption.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (147 words)
Cancer therapy-induced thrombocytopenia (CTIT) is a common complication in patients with solid tumors during their cancer treatment. The sole therapeutic agents approved for this indication are recombinant human interleukin-11 (rhIL-11) or recombinant human thrombopoietin (rhTPO). Romiplostim-N01 is a novel thrombopoietic agent launched in China in 2024, which has demonstrated preliminary therapeutic efficacy in the management of CTIT. This retrospective study compared romiplostim-N01 with rhIL-11 or rhTPO for CTIT management. Ninety-two matched consecutive patients with grade ≥2 CTIT (platelet [PLT] count .05). The median duration of chemotherapy delay was significantly shorter in the romiplostim-N01 group compared with the rhIL-11/rhTPO group (5.5 vs 9.5 days, P < .001). Multivariate regression analysis confirmed that romiplostim-N01 was independently associated with an elevation in PLT count. These findings position romiplostim-N01 as a favorable alternative to rhIL-11 or rhTPO in CTIT, given its enhanced early PLT response and reduced risk of chemotherapy disruption.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 42216387 · DOI · PMC13225505
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article Platelets [5]
- Research article Lineage-specific hematopoietic growth factors [6]
- Research article Thrombopoietin receptor agonists: ten years later [7]
- Research article Thrombopoietin [8]
- Research article Recombinant human thrombopoietin: basic biology and evaluation of clinical studies [9]
- Research article Thrombocytopenia in chronic liver disease [10]
References
- Park HJ, Li J, Hannah R, Biddie S, Leal-Cervantes AI, Kirschner K, et al.. Cytokine-induced megakaryocytic differentiation is regulated by genome-wide loss of a uSTAT transcriptional program. . 2016. PMID 26702099 · DOI · PMC4801948
- Haetscher N, Feuermann Y, Wingert S, Rehage M, Thalheimer FB, Weiser C, et al.. STAT5-regulated microRNA-193b controls haematopoietic stem and progenitor cell expansion by modulating cytokine receptor signalling. . 2015. PMID 26603207 · DOI · PMC4674773
- Pecci A, Ragab I, Bozzi V, De Rocco D, Barozzi S, Giangregorio T, et al.. Thrombopoietin mutation in congenital amegakaryocytic thrombocytopenia treatable with romiplostim. . 2018. PMID 29191945 · DOI · PMC5760853
- Yu T, Xiong Z, Su Y, Jian Y, Liu L, Wang Y. Romiplostim-N01 for cancer therapy-induced thrombocytopenia: A propensity score-matched cohort study. . 2026. PMID 42216387 · DOI · PMC13225505
- George JN. Platelets. Lancet. 2000. PMID 10801186 · DOI
- Kaushansky K. Lineage-specific hematopoietic growth factors. N Engl J Med. 2006. PMID 16687716 · DOI
- Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB. Thrombopoietin receptor agonists: ten years later. Haematologica. 2019. PMID 31073079 · DOI · PMC6545830
- Kaushansky K. Thrombopoietin. N Engl J Med. 1998. PMID 9731092 · DOI
- Kuter DJ, Begley CG. Recombinant human thrombopoietin: basic biology and evaluation of clinical studies. Blood. 2002. PMID 12411315 · DOI
- Peck-Radosavljevic M. Thrombocytopenia in chronic liver disease. Liver Int. 2017. PMID 27860293 · DOI
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-31.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.