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Home › Conditions › Thrombocytopenia

Thrombocytopenia

Subtypes of Thrombocytopenia

Neonatal thrombocytopenia 1 Kasabach-Merritt phenomenon 0

3 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Thrombocytopenia

36 trials tracked for Thrombocytopenia: 10 in phase 3 or 4 and 4 with published results. The most-cited published study has 157 citations.

36Trials tracked
10Phase 3 & 4
0Recruiting
4With published results
Phase distribution
Phase 4 1 Phase 3 9 Phase 2 17 Phase 1 3 Other / NA 6
  1. Phase 3 Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients Completed · 157 cited
  2. Phase 3 Prospective Study of Patients With Thrombocytopenia Following HSCT Completed · 137 cited
  3. Phase 3 Safety and Efficacy Study of Lusutrombopag for Thrombocytopenia in Patients With Chronic Liver Disease Undergoing Elective Invasive Procedures Completed · 14 cited
  4. Phase 3 American Trial Using Tranexamic Acid in Thrombocytopenia Completed · 9 cited
  5. Phase 4 Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP) Completed
  6. Phase 3 Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy Completed
Show 30 more trials
  1. Phase 3 Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura Completed
  2. Phase 3 Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Completed
  3. Phase 3 An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP Completed
  4. Phase 3 Optimal Platelet Dose Strategy for Management of Thrombocytopenia Completed
  5. Phase 2 Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS) Completed
  6. Phase 2 Study of Drug to Reduce Thrombocytopenia in Patients Receiving Chemo for Ovarian, Fallopian Tube or Peritoneal Cancer Completed
  7. Phase 2 Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents Completed
  8. Phase 2 Study to Evaluate Safety, Tolerability and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia Completed
  9. Phase 2 Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) Completed
  10. Phase 2 Chronic Hepatitis C Virus Related Thrombocytopenia to Evaluate the Effects of E5501 Completed
  11. Phase 2 Dose/ Schedule Finding Trial of Romiplostim for Chemotherapy-Induced Thrombocytopenia (CIT) in Non-Small Cell Lung Cancer (NSCLC) Completed
  12. Phase 2 A Safety and Efficacy Study to Evaluate AMG 531 Treatment in Subject With Myelodysplastic Syndrome Receiving Revlimid Completed
  13. Phase 2 A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients Completed
  14. Phase 2 A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) Completed
  15. Phase 2 Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia Completed
  16. Phase 2 Differentiation Therapy With Decitabine in Treating Patients With Myelodysplastic Syndrome Completed
  17. Phase 2 Ex Vivo-activated Autologous Lymph Node Lymphocytes in Treating Patients With Chronic Lymphocytic Leukemia Completed
  18. Phase 2 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia Completed
  19. Phase 2 Eltrombopag for Post Transplant Thrombocytopenia Completed
  20. Phase 2 A Dose and Schedule Finding Trial With AMG 531 for Chemotherapy Induced Thrombocytopenia (CIT) in Adults With Lymphoma Completed
  21. Phase 2 Lenalidomide and Ofatumumab in Treating Participants With Previously Treated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma Completed
  22. Phase 1 Phase I Study of Eltrombopag for Promoting Thrombopoiesis After Total Body Irradiation Completed
  23. Phase 1 Thrombosomes® in Bleeding Thrombocytopenic Patients Completed
  24. Phase 1 Phase 1 Safety Study of Dimethyl Sulfoxide Cryopreserved Platelets Completed
  25. N/A Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS) Completed
  26. N/A Study of the Effect of Differing Platelet Transfusion Times in Neonates Completed
  27. N/A XIENCE 28 USA Study Completed
  28. N/A Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Completed
  29. N/A XIENCE 28 Global Study Completed
  30. N/A Platelet Count Trends in Pre-eclamptic Parturients Completed

Showing the 36 most-cited and recently-updated of 36 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Thrombocytopenia: what the trials found

Romiplostim has demonstrated significant efficacy in improving platelet responses. Clinical trials showed a substantial increase in the percentage of participants achieving a durable platelet response (52.4% vs. 10.0%, p=0.0018) and an overall platelet response (71.4% vs. 20.0%, p=0.0002), alongside a significant increase in the number of weeks with a platelet response (12.0 vs. 1.0, p=0.0004) 2. Additionally, Romiplostim was associated with a positive change in quality of life scores (EQ-5D index) 9.

Lusutrombopag showed high efficacy rates compared to its comparator, with 64.8% of participants requiring no platelet transfusion prior to primary procedures and no rescue therapy for bleeding within 7 days post-procedure, compared to only 29.0% in the comparison group (p<0.0001). Similarly, 63.0% of patients treated with Lusutrombopag required no platelet transfusions during the study period versus 29.0% in the control group (p<0.0001) 4.

Eltrombopag was evaluated across multiple trials to assess its role in treatment and safety, including monitoring for grade 2 or higher bleeding events [13, 14, 17]. In a comparative study of medical standards of care for ITP, significant differences were observed in treatment failure rates and the necessity of splenectomy over a 52-week period (p<0.0001 and p=0.0005 respectively) 7.

Decitabine was evaluated in clinical settings with reported data on non-hematologic toxicity, where 16 patients experienced > Grade 2 non-hematologic toxicity 22. Regarding transfusion management, medium dose prophylactic platelet transfusions were associated with significantly lower rates of platelet transfusion episodes compared to other protocols (p<0.001) and lower overall platelet utilization (p=0.02) 10.

Recent results — preliminary, needs further review

  • Avatrombopag showed a statistically significant difference in the number of participants achieving a platelet response (≥100 x 10^9/L) by day 21 (p=0.0236), though this finding is currently not corroborated 18.
  • Placebo-controlled studies showed significantly lower annualized rates of platelet transfusion events (p<0.001) and overall bleeding events (p=0.026) in the intervention group compared to placebo, though results for clinically significant bleeding events were not statistically significant (p=0.13) 20.

For the clinician treating this condition

  • Romiplostim is associated with significantly higher rates of durable and overall platelet responses compared to standard interventions.
  • Lusutrombopag demonstrates a high rate of patients requiring no platelet transfusions before primary procedures or during the study period.
  • Prophylactic platelet transfusion strategies can significantly reduce the number of transfusion episodes and total platelet utilization.

AI synthesis of 8 cited trials, updated Jun 23, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Mechanistic-driven diagnosis and therapy shift focus from platelet counts to underlying pathophysiology of thrombocytopenia
Hematology Sys. Review
Mechanistic-driven diagnosis and therapy shift focus from platelet counts to underlying pathophysiology of thrombocytopenia New Framework Offers Precise Treatment for Low Blood Platelets
This systematic review synthesizes the multidimensional pathogenesis of thrombocytopenia across five axes including production, immune-media…
A new diagnostic framework moves beyond simple blood counts to identify the specific cause of low platelets for more precise treatment.
Frontiers Sep 29, 2026
Restrictive platelet thresholds reduce transfusions without increasing death or bleeding risk in thrombocytopenic neonates
Neurology Sys. Review
Restrictive platelet thresholds reduce transfusions without increasing death or bleeding risk in thrombocytopenic neonates Restrictive transfusion thresholds reduce blood use in newborns without increasing death risk
A systematic review of 856 neonates found that restrictive platelet transfusion thresholds significantly reduced transfusion use. Crucially,…
Newborns with low platelet counts get fewer transfusions when doctors use stricter rules, saving blood without raising death or bleeding ris…
Cochrane May 21, 2026
Case report describes thrombocytopenia in two siblings after elemental mercury vapor exposure
Psychiatry
Case report describes thrombocytopenia in two siblings after elemental mercury vapor exposure Two siblings developed low platelet counts after exposure to mercury vapor
A case report describes two siblings who developed thrombocytopenia following elemental mercury vapor exposure.
Two siblings developed low platelet counts after breathing mercury vapor, adding a blood-related risk to known mercury dangers.
CDC Apr 3, 2026