November 2025: New In Coagulation
by Donna Castellone • November 04, 2025
Our Monthly complilation of the latest studies, guidelines and discussions in coagulation.
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Essential Thrombocythemia: New Standard-of-Care Option?
The initial therapy for essential thrombocythemia (ET) is hydroxyurea however some patients fail to respond. ET is a myeloproliferative neoplasm that caused the over production of abnormal platelets. A new interferon drug ropeginterferon alfa-2b (ropeg) showed significantly improved responses and other important outcomes compared with the platelet-reducing agent anagrelide. Ropeg is approved for polycythemia vera (PV) which is also a Philadelphia chromosome negative-disorder like ET. The research may lead to an additional indication in ET and would be the first approved new option since anagrelide. Up to a third of patients with ET are either intolerant or fail to respond to the standard hydroxyurea. Anagrelide was approved in 1997 as a second-line agent, however no new treatment has been approved since then.
The 3 SURPASS-ET trial enrolled 174 patients with ET that were intolerant or refractory to hydroxyurea. Patients in the ropeg group (n = 91, median age 61, 50% female) were treated with a starting dose of 250 mcg, titrating to 350 mcg at week 2 and then, if tolerated, to 500 mcg from week 4 every 2 weeks. The anagrelide group had a median age of 64 with half female. The primary endpoint was a response to the EU LeukemiaNet criteria resulting in a 10 point decrease in the Myeloproliferative Neoplasms Symptom Assessment Form Total Symptom Score for at least 12 weeks, normalization of peripheral blood counts for at least 12 weeks, absence of vascular events and disease progression, and disappearance of bone marrow histological abnormalities. This was met by 42.9% of the ropeg group versus 6% in the anagrelide group at 9 and 12 months. The platelet count response was met by 56% in the ropeg group versus 21.7% in the anagrelide group, white blood cell count response was achieved in 73.6% vs 13.3%, and peripheral blood count remission was achieved in 56% vs 6.0%. Nonprogression of splenomegaly occurred in 87.9% and 54.2% of ropeg and anagrelide patients, respectively, and an absence of hemorrhagic or thrombotic events occurred in 84.6% and 51.8% of patients, respectively.
Grade 3 or higher adverse events occurred in 23.1% in the ropeg group vs 33.8% in the anagrelide group, and serious adverse events occurred in 15.4% vs 30.0%. Adverse event-related discontinuations occurred in just 5.5% in the ropeg group vs 20.0% in the anagrelide group. SURPASS ET provides now phase 3 data proving the safety, efficacy, and impact on variant allele frequency for ET in the second line.
EMA Endorses Generic Blood Thinner in New Formulation
Rivaroxaban Koanaa received a favorable recommendation from the EMA as a new generic oral anticoagulant. A generic version of Xarelto has been authorized in the EU in 2008. These drugs are used in the prevention of VTE and treatment and prevention of DVT and PE, prevention of stroke and systemic embolism in adults with AF. This is available as orodispersible films, broadening administration options for patients with difficulty swallowing tablets. This drug works as a direct factor Xa inhibitor which disrupts the intrinsic and extrinsic coagulation pathways preventing thrombin generation and clot formation.
Different strengths are used for different indications. A dose of 10 mg is used for prevention of VTE in adults undergoing elective hip or knee replacement surgery. While 15mg is used in adults for prevention of stroke and systemic embolism in nonvalvular atrial fibrillation with risk factors such as heart failure, hypertension, diabetes, age ≥ 75 years, or prior stroke/transient ischemic attack. It is also used in adults for treatment and prevention of recurrent DVT and PE. This dose is also suggested for children or adolescents < 18 years weighing 30-50 kg: Treatment and prevention of VTE after at least 5 days of parenteral anticoagulation. A dose of 20 mg has the same adult indication as 15mg, while in children or adolescents < 18 years weighing > 50 kg: Treatment and prevention of VTE after at least 5 days of parenteral anticoagulation.
High Incidence of Thromboembolic Events in New-Onset SLE with Antiphospholipid Syndrome
A population based study that looked at 700 patients (mean age 39, 85% female) was conducted in Norway with an 8 year follow up. In patients that have new onset systemic lupus erythematosus (SLE) 11% had developed APS, with 35% occurring within the first year of diagnosis. In 156 patients up to 23% experience one or more thrombotic events and 89 experienced their first event after SLE diagnosis. Those diagnosed with antiphospholipid antibody positive were highest during the first year and declined after the subsequent 4 years. The probability of remaining free of TEs was significantly lower in patients who were positive vs negative for antiphospholipid antibody at 1 year after SLE diagnosis. An increased risk for a thrombotic event was seen in males, older age, smoking, hemolytic anemia and more than one APA positive test. Limitations of the study included the study design did not infer causality.
Anticoagulant Alone Safer in Stroke Patients With Afib and Atherosclerosis
The ATIS-NVAF (Antithrombotic Therapy in Ischemic Stroke Patients with Non-Valvular Atrial Fibrillation and Atherothrombosis) included 316 patients from 41 centers in Japan. Patients had a mean age of 77.2 ears and 28.5% were female. Patients were randomized to receive an anticoagulant plus an antiplatelet or anticoagulant monotherapy. Choice of drug was decided by the physician. 94% received DOACs, most often apixaban., Antiplatelet therapy included aspirin (52%), clopidogrel (31%), or cilostazol (Pletal, 17%). Results showed that adding an antiplatelet to anticoagulant therapy increased bleeding without preventing more ischemic events. Primary endpoint included a composite of ischemic and major bleeding events. This occurred in 17.8% of the combination group, and 19.6% of those on monotherapy. While ischemic cardiovascular events, a secondary outcome didn't significantly differ between groups o. Occurrence was seen in 11.1% of combination therapy versus 14.2% on anticoagulant therapy. However, bleeding was more common in combination therapy, 19.5% versus 8.6%. Ischemic stroke or TIA occurred within 8 to 360 days of onset of nonvalvular AF and one type of atherosclerotic CVD. Limitations of this trial include open label design, clinical selection of treatment and the Japanese population which may limit generalizability.
Low-Dose Aspirin: Cancer Prevention Option for Older Adults?
Aspirin has been labeled as a promising agent for colorectal cancer (CRC) prevention; low doses can reduce CRC risk as well as recurrence rates. In a secondary analysis of the ASPREE study it was found that aspirin can be effective for some though not for others. The analysis showed heterogenicity in aspirin results. In groups that were divided into treatment favorable and unfavorable subgroups, low-dose aspirin was associated with a lower cancer risk in the favorable subgroup but a greater cancer risk in the unfavorable subgroup.
The secondary analysis identified subgroups that were more likely to experience a benefit or harm from low dose aspirin for cancer prevention to use for guidance in personalized prevention strategies. There were 9350 randomized participants to either low dose aspirin or placebo. Those associated with aspirin benefit included older, nonsmoker, family history or cancer and lower BMI. The strongest predictor was the presence of clonal hematopoiesis of indeterminate potential (CHIP) with a variant allele frequency of 10% or greater.
CHIP is an age-related phenomenon where blood-forming cells, known as hematopoietic stem cells, acquire genetic mutations, which have been linked to an increased risk of developing cardiovascular disease and blood cancers.
Using a validated predictive model, the researchers divided participants into treatment-favorable and unfavorable subgroups to predict the personalized treatment effect of low-dose aspirin on cancer incidence.
The favorable subgroup was 59.1% versus 40.9% who were in the unfavorable treatment subgroup. Low dose aspirin was associated with a reduced cancer risk in the favorable group but a greater risk in the unfavorable group.
D-dimer reporting, INR among the variables that matter
Protocols are needed to handle anticoagulant interferences in coagulation testing. Heparins, Vitamin K antagonists and DOAC (IIa and Xa) can be problematic. LA has been known to cause false positives or false negatives. A process is required in the laboratory to identify the anticoagulant. This can be done by checking thrombin time, anti-Xa or INR. If there is a measurable anti- Xa it is possible to neutralize the heparin prior to testing, if there is still anti-Xa, it may suggest the presence of a DOAC. Some assays, like DRVV, may neutralize up to 1U of heparin. The TT is very sensitive not only to heparin but also to a DTI. Also, novel anti-hemophila medication can not be measured or monitored.
Post analytical variables include the INR and verification. CAP requires laboratories to verify the calculation of the INR with each ISI reagent change, as well as the calculations used for patient results. This is important with INR in the range of 2-3.
Units of the D-dimer are another post analytical variable. There are 2 units that can be used- DDU or FEU. FEU = DDU x2 and DDU= 0.5 x FEU. The reporting unit should match what is stated by the manufacturer. If different units are used it must be verified annually. Additionally, if the DD is used in the evaluation of VTE, the manufacturer cutoff should be used. If the DD is used for DIC a reference interval is u sed. It is important to know if the test is quantitative or used for exclusion. There is no DD standard.
Benefit of Dual Antiplatelet Therapy Murky in Valve-in-Valve Intervention
A major adverse cardiac and CVE occurred in 5.6% of patients receiving DAPT versus 7.5% of patients who received single antiplatelet therapy (SAPT). However the incidence of stroke was lower in DAPT (0.6%) versus SAPT (4.6%). There was increased bleeding risk in DAPT (9.8% vs 3.7%), but most bleeds were minor and there was no difference in major bleeds between treatments. Most DAPT bleeds occurred within the first few days post TAVI.
Using DAPT post valve-in-valve TAVI results from the higher thrombotic risk associated with bioprosthetic valve failure. One finding in DAPT's favor was the unchanged mean transvalvular gradient 1 year after valve-in-valve TAVI compared with the +2 mm Hg median increase in SAPT patients. The gradient increase in patients treated with SAPT suggests that subclinical valve thrombosis may represent the underlying mechanism.
This was an observational study from data from 10 centers in which they compared 113 SAPT (aspirin) users and 165 peers given DAPT ( clopidogrel & aspirin), patients on OAC were excluded. The DAPT group was older (81 vs 78 years) with a higher surgical risk along with a disproportionate number of cases of PCI (42.2% vs 5.3%). Valve-in-valve TAVI was performed with a mix of self-expanding and balloon-expandable valves. DAPT recipients were more likely to have gotten a balloon-expandable valve (50.3% vs 27.4%).
There was no difference in the groups in the type of degenerative valve or the valve failure mechanism. Post valve-in-valve TAVI the average postprocedural transvalvular gradients were higher in the DAPT group, while the incidence of moderate or severe paravalvular regurgitation was comparable between groups. The results may have selection bias as a study limitation.
VTE and DVT and DOACs, Oh My! A PCP Guide to Anticoagulation
The big two DOACs seen in clinical practice are apixaban and rivaroxaban. In deciding what medication to use on a patient it may be helpful to look at the patients' renal function. If they present with diminished renal function apixaban may be a better choice. The next thing that needs to be considered is the patients' bleeding risk. If the risk is high, apixaban should be considered. Findings from the COBRA trial shows that apixaban has a lower risk of bleeding complications. Dabigatran has seemed to fall out of favor due to GI upset and intolerance of the medication and a lead-in period is required for a parenteral medication.
For patients on a prolonged trip who are not on long term anticoagulation with a history of VTE, a low dose of DOAC may be used such as apixaban (2.5 mg) or rivaroxaban (10 mg), just as a prophylactic dose before travel in order to hopefully reduce their risk.
Another area of concern is distal DVT, some ultrasonographers will just do proximal but others will go down into the calf to find a distal DVT. You can either follow it to see if it will spontaneously resolve or put the patient on anticoagulation for several months. Guidelines would suggest to treat it, but you wouldn't have found it unless the patient was symptomatic.
Regarding reversal agents for DOAC, if a patient has bleeding should andexanet alfa be used? Studies that have looked at this have seen an increased risk of thromboembolic events and it is causing clots as opposed to reversing bleeding. It is expensive and by the time the order is released from the pharmacy it may have taken 6-8 hours at which point it may not be worth it, based on when the patient had their last dose. Adverse events such as thrombotic complications have been seen. Several clinicians have returned to using prothrombin complex until more information can be obtained about the direct reversal agent.
FDA Slaps Boxed Warning on Bleeding Control Drug
Due to errors of inadvertent neuraxial administration of tranexamic acid (TXA) the FDA is requiring stronger box warnings. The drug was administered neuraxially instead of the intended local anesthetic resulting in prolonged hospitalization and death. As a result the FDA is requiring stronger warnings to avoid medication errors. The boxed warning reads:
“Cyklokapron is for intravenous use only. Serious, including fatal, adverse reactions including seizures and cardiac arrhythmias have occurred when Cyklokapron was inadvertently administered intrathecally via the neuraxial route."
TXA was stored next to local anesthetics and the product was not verified prior to administration, resulting in these errors. It will also state that it is only to be administered by IV. These drugs should not be stored next to local anesthetics.
TXA is used to prevent the breakdown of blood clots and to prevent excessive bleeding, and to reduce or prevent hemorrhage as well as reducing the need for replacement therapy following tooth extraction.
JOURNAL CLUB
Clonal Hematopoiesis and Cardiovascular Disease and Bleeding Risk and the Effectiveness of Aspirin
Zoe K. McQuilten, MBBS, PhD; Le T. P. Thao, PhD; Alexander G. Bick, MD, PhD, et al.
JAMA Cardiol, Published Online: October 15, 2025
Abstract
Importance Clonal hematopoiesis of indeterminate potential (CHIP) has been associated with increased risk of cardiovascular disease (CVD) events and mortality. However, there are no approved therapies for preventing or treating CHIP.
Objective To investigate whether low-dose aspirin might benefit older adults with CHIP for the primary prevention of CVD.
Design, Setting, and Participants This was a prespecified substudy of the Aspirin in Reducing Events in the Elderly (ASPREE) double-blind, randomized clinical trial of daily low-dose aspirin vs placebo evaluating disability-free survival, which took place at primary and community care facilities in the US and Australia. Enrollment was from March 2010 to December 2014, and the randomized trial ended in June 2017. Community-dwelling Australian adults aged 70 years and older without a diagnosed cardiovascular event, atrial fibrillation, a serious intercurrent illness likely to cause death within the next 5 years, anemia, or a current or recurrent condition with a high risk of bleeding were included in the original study. Of 19 114 in the original trial, 11 402 were included in the substudy, and 9434 were included in the analysis. Follow-up for this substudy went through June 2022, with data analysis in February 2025. In-trial median (IQR) follow-up time was 4.6 (3.5-5.6) years, and posttrial observational follow-up was 8.7 (7.5-10.1) years from randomization.
Interventions Participants were randomized to aspirin, 100 mg, daily or placebo.
Main Outcomes and Measures CHIP was measured in blood specimens collected at trial entry. Major adverse cardiovascular events (MACEs), including fatal and nonfatal ischemic stroke, nonfatal myocardial infarction and coronary heart disease death, and clinically significant bleeding were adjudicated by independent expert committees blinded to trial-group assignments.
Results A total of 9434 participants (median [IQR] age, 73.7 [71.6-77.1] years; 5067 [54%] female) provided a sample at baseline for analysis, 2124 of whom (23%) had CHIP at variant allele fraction (VAF) ≥2%, with 532 (5.6%) at ≥10% VAF. CHIP was not associated with increased risk of MACEs at 2% to 10% VAF (adjusted hazard ratio [aHR], 0.84, 95% CI, 0.68-1.03;
Conclusion and Relevance In this secondary analysis of a randomized clinical trial of daily low-dose aspirin in healthy adults 70 years and older, CHIP was not associated with higher CVD risk. However, participants with CHIP had a greater risk of clinically significant bleeding. There was no evidence that participants with CHIP were more likely than those without CHIP to benefit or experience more harm from aspirin when used for primary prevention of CVD events.
Long-Term Anticoagulation Discontinuation After Catheter Ablation for Atrial Fibrillation:
The ALONE-AF Randomized Clinical Trial
Daehoon Kim, MD; Jaemin Shim, MD; Eue-Keun Choi, MD, et al,
JAMA, Published Online: August 31, 2025
Abstract
Importance Data from randomized clinical trials on a long-term anticoagulation strategy for patients after catheter-based ablation for atrial fibrillation (AF) are lacking.
Objective To evaluate whether discontinuing oral anticoagulant therapy provides superior clinical outcomes compared with continuing oral anticoagulant therapy in patients without documented atrial arrhythmia recurrence after catheter ablation for AF.
Design, Setting, and Participants A randomized clinical trial including 840 adult patients (aged 19-80 years) who were enrolled and randomized from July 28, 2020, to March 9, 2023, at 18 hospitals in South Korea. Enrolled patients had at least 1 non–sex-related stroke risk factor (determined using the CHA2DS2-VASc score [range, 0-9]) and no documented recurrence of atrial arrhythmia for at least 1 year after catheter ablation for AF. The CHA2DS2-VASc score is used as an assessment of stroke risk among patients with AF (calculated using point values for congestive heart failure, hypertension, ≥75 years of age, diabetes, stroke or transient ischemic attack, vascular disease, between 65 and 74 years of age, and sex category). The date of final follow-up was June 4, 2025.
Interventions The patients were randomly assigned in a 1:1 ratio to discontinue oral anticoagulant therapy (n = 417) or continue oral anticoagulant therapy (with direct oral anticoagulants; n = 423).
Main Outcomes and Measures The primary outcome was the first occurrence of a composite of stroke, systemic embolism, and major bleeding at 2 years. Individual components of the primary outcome (such as ischemic stroke and major bleeding) were assessed as secondary outcomes.
Results Of the 840 adults randomized, the mean age was 64 (SD, 8) years, 24.9% were women, the mean CHA2DS2-VASc score was 2.1 (SD, 1.0), and 67.6% had paroxysmal AF. At 2 years, the primary outcome occurred in 1 patient (0.3%) in the discontinue oral anticoagulant therapy group vs 8 patients (2.2%) in the continue oral anticoagulant therapy group (absolute difference, –1.9 percentage points [95% CI, −3.5 to −0.3];
Conclusions and Relevance Among patients without documented atrial arrhythmia recurrence after catheter ablation for AF, discontinuing oral anticoagulant therapy resulted in a lower risk for the composite outcome of stroke, systemic embolism, and major bleeding vs continuing direct oral anticoagulant therapy.
Intra-Arterial Alteplase After Successful Endovascular Reperfusion in Acute Stroke
The PEARL Randomized Clinical Trial
JAMA, Published Online: October 13, 2025
doi: 10.1001/jama.2025.16876
Abstract
Importance Functional outcomes in patients with acute ischemic stroke due to large-vessel occlusion who undergo thrombectomy remain suboptimal, and the benefits of intra-arterial alteplase after thrombectomy remain uncertain.
Objective To investigate whether treatment with intra-arterial alteplase after successful endovascular reperfusion improves functional outcomes among patients with acute, anterior-circulation, large-vessel occlusion stroke.
Design, Setting, and Participants This multicenter, randomized clinical trial recruited patients with anterior-circulation, large-vessel occlusion stroke within 24 hours of symptom onset who achieved successful reperfusion (expanded Thrombolysis in Cerebral Infarction scale score of ≥2b50) after thrombectomy. Guideline-based intravenous thrombolysis was allowed. Patients were randomized between August 1, 2023, and October 16, 2024, and the trial was conducted at 28 hospitals in China. Final follow-up occurred on January 7, 2025.
Interventions Intra-arterial alteplase treatment (n = 164) with 0.225 mg/kg (maximum dose of 20 mg) vs standard treatment (n = 160).
Main Outcomes and Measures The primary outcome was the proportion of patients with a modified Rankin Scale score of 0 or 1 at 90 days (score range, 0 [no symptoms] to 6 [death]; a score of 0 or 1 indicates an excellent outcome). The safety outcomes included symptomatic intracranial hemorrhage within 36 hours of randomization, all-cause mortality within 90 days, and any intracranial hemorrhage within 36 hours.
Results Of the 324 patients randomized (median age, 68 years [IQR, 58-75 years]; 99 were female [30.6%]), 1 patient in each group was lost to follow-up. The proportion of patients with a modified Rankin Scale score of 0 or 1 at 90 days was 44.8% (73/163) in the intra-arterial alteplase group vs 30.2% (48/159) in the standard treatment group (adjusted risk ratio [RR], 1.45 [95% CI, 1.08-1.96];
Conclusions and Relevance Among patients with acute, anterior-circulation, large-vessel occlusion stroke who achieved successful endovascular reperfusion by mechanical thrombectomy, intra-arterial alteplase resulted in a higher likelihood of excellent outcomes at 90 days. The incidence of all-cause mortality and any intracranial hemorrhage was higher in patients who received intra-arterial alteplase, although these differences were not statistically significant.
Age-Stratified Effect of Rivaroxaban Monotherapy for Atrial Fibrillation in Stable Coronary Artery Disease:
A Post Hoc Analysis of the AFIRE Randomized Clinical Trial
Junichi Yamaguchi, MD, PhD; Hiroyuki Arashi, MD; Nobuhisa Hagiwara, MD, PhD, et al,
JAMA Cardiol, Published Online: August 13, 2025
Abstract
Importance Antithrombotic therapy is crucial for older patients with coronary artery disease (CAD) and atrial fibrillation (AF) who are at a high risk of bleeding and thrombotic events.
Objective To examine the age-stratified effects of rivaroxaban monotherapy compared with those of rivaroxaban plus antiplatelet agent combination therapy.
Design, Setting, and Participants This was a post hoc secondary analysis of the Atrial Fibrillation and Ischemic Events With Rivaroxaban in Patients With Stable Coronary Artery Disease (AFIRE) open-label randomized clinical trial. This was a multicenter study conducted in Japan from February 23, 2015, to July 31, 2018. Patients with AF and stable CAD who had undergone percutaneous coronary intervention or coronary artery bypass grafting 1 or more years earlier or who had angiographically confirmed CAD that did not require revascularization were enrolled. Participants were stratified into 4 groups by age (<70 years, 70-74 years, 75-79 years, and ≥80 years). Study data were analyzed from August 2024 to July 2025.
Interventions Rivaroxaban monotherapy or rivaroxaban plus antiplatelet agent therapy.
Main Outcomes and Measures The primary efficacy end point was a major adverse cardiovascular event, defined as a composite of stroke, systemic embolism, myocardial infarction, unstable angina requiring revascularization, or death from any cause. The primary safety end point was major bleeding.
Results This study included a total of 2215 participants (mean [SD] age, 74.3 [8.2] years; 1751 male [79.1%]). The incidence of primary efficacy end points per patient-year for rivaroxaban monotherapy vs rivaroxaban plus antiplatelet agent therapy was 3.2% vs 4.3% (<70 years), 3.2% vs 2.8% (70-74 years), 3.8% vs 5.3% (75-79 years), and 6.2% vs 10.3% (≥80 years). The hazard ratios were 0.74 (95% CI, 0.40-1.37) for those younger than 70 years, 1.16 (95% CI, 0.55-2.45) for those aged 70 to 74 years, 0.72 (95% CI, 0.41-1.26) for those aged 75 to 79 years, and 0.61 (95% CI, 0.40-0.93) for those 80 years and older (
Conclusions and Relevance Results of this post hoc analysis of the AFIRE randomized clinical trial reveal that rivaroxaban monotherapy reduced the risk of major cardiovascular events and major bleeding across the broad range of age in patients with AF and stable CAD. Possible age-related differences in trends, with more pronounced efficacy in older patients and more pronounced safety in younger patients, should be considered as hypothesis generating and require further research.
Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment:
The PINES Randomized Clinical Trial
Kristin A. Shimano, MD; Amanda B. Grimes, MD; Shipra Kaicker, MD, et al,
JAMA, Published Online: October 22, 2025
Abstract
Importance Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. Efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia is unknown.
Objective To determine if the proportion of patients with a platelet response is significantly greater in patients with newly diagnosed immune thrombocytopenia treated with eltrombopag than in those treated with standard therapy (first-line treatments).
Design, Setting, and Participants This phase 3, randomized clinical trial enrolled patients (aged 1-<18 years) with newly diagnosed primary immune thrombocytopenia (platelet count <30 × 109/L who required pharmacological treatment but did not have severe bleeding or need a rapid increase in platelet count) from May 7, 2019, to January 25, 2024, at 23 centers participating in the Pediatric ITP Consortium of North America. Final follow-up occurred on February 26, 2025.
Interventions Eltrombopag was administered orally based on a standard dosing schedule (n = 78) vs standard therapy (investigator choice of glucocorticoids, intravenous immunoglobulin, or anti-D immunoglobulin) (n = 40).
Main Outcomes and Measures The primary outcome was a sustained platelet response defined as 3 or more of 4 platelet counts greater than 50 × 109/L during weeks 6 to 12 without rescue treatment. The secondary outcomes included bleeding scores, change in health-related quality of life, and serious adverse events.
Results Of 118 pediatric patients (median age, 8 years [IQR, 4-12 years]; 49% were male), 63% experienced an initial treatment failure after observation or medical therapy. Enrollment ended after a planned interim analysis met a prespecified threshold for efficacy. Of 71 patients in the eltrombopag group, 46 (65% [95% CI, 54%-76%]) had a sustained platelet response compared with 13 of 37 patients (35% [95% CI, 20%-51%]) in the standard therapy group (between-group difference, 30% [95% CI, 11%-49%];
Conclusions and Relevance In pediatric patients with newly diagnosed immune thrombocytopenia requiring pharmacological treatment, eltrombopag resulted in a higher rate of sustained platelet response compared with standard therapy. Eltrombopag may be an effective option for pediatric patients with newly diagnosed immune thrombocytopenia with nonsevere bleeding who warrant medical intervention.