About

Alexis A. Thompson, MD, MPH, is a Professor of Pediatrics (Hematology) and the Chief of the Division of Hematology at the Children's Hospital of Philadelphia. She holds the Elias Schwartz MD Endowed Chair in Hematology at the Children's Hospital of Philadelphia. Her department is the Department of Pediatrics, specifically within the Division of Hematology. Dr. Thompson's educational background includes a degree from Pomona College (1979), an MD from Tulane University School of Medicine (1983), and an MPH in Health Services from the University of California, Los Angeles (1987). Her professional focus is on pediatric hematology, with particular expertise in sickle cell disease and related hematologic conditions. She has contributed extensively to research in this field, including studies on pain management, cortisol levels, gene therapy, and the safety of treatments for sickle cell disease.

Research topics

• Medicine
• Computer Science
• Internal medicine
• Data science
• Pediatrics
• Nursing
• Economic growth
• Family medicine
• Economics

Selected publications

• Secretory phospholipase A2 (sPLA2) during vaso-occlusive pain episodes (VOE) and acute chest syndrome (ACS) in sickle cell disease (SCD): Results from a prospective multi-center randomized controlled trial (RCT)

  Blood · 2025-11-03

  articleOpen access

  Abstract Background: Acute chest syndrome (ACS) is the leading cause of morbidity and mortality in children with SCD, often developing during vaso-occlusive episodes (VOE) with minimal or no early clinical signs. Most children present with a normal lung exam, making prompt detection challenging. Secretory phospholipase A2 (sPLA2), a potent inflammatory mediator, has been shown to rise prior to ACS onset and may aid in predicting its development and severity in the acute setting. Objective: Assess sPLA2 as a marker for ACS risk and severity in patients with SCD hospitalized for VOE Methods: Cross-sectional analysis of data from a multicenter, double-blind, placebo-controlled, phase-3 RCT (NCT04839354) evaluating intravenous arginine therapy in patients aged 3–21 years with SCD-VOE conducted at 10 pediatric emergency departments (ED) in the US, endorsed by the Pediatric Emergency Care Applied Research Network (PECARN). Plasma sPLA2 levels at ED presentation, hospital day 2, and on the day of hospital discharge (DC) were analyzed via enzyme-linked immunosorbent assay (ELISA). sPLA2 level ≥48 ng/mL is considered elevated based on an established SCD-specific cutoff. ACS severity was classified as mild (no oxygen/transfusion), moderate (required oxygen/transfusion), or severe (required BIPAP, intubation, or PICU admission). Vital signs and clinical labs were analyzed. Results: Overall 271 patients were randomized, and 251 had sufficient blood samples for sPLA2 assessment at presentation and were included in the analysis (mean age 14±4 years; 53% male, 73% HbSS/Sβ°, and 75% on hydroxyurea). Mean plasma sPLA₂ level at ED presentation was 116±131 ng/mL, with elevated levels ≥48 ng/mL in 60% of patients with VOE. ACS was diagnosed in 21% of patients enrolled (n=52), with 18 diagnosed in the ED and 34 were later diagnosed with ACS during admission. Although the majority of ACS patients (85%) had a normal respiratory exam at ED presentation, 71% had elevated sPLA2 levels ≥48 ng/mL. Patients diagnosed with ACS at any point during enrollment (n=52) had significantly higher mean plasma sPLA2levels (n=52) at ED presentation compared to patients with no ACS (n=199) (152±147 vs 107±126 ng/mL, p=0.01). Moreover, patients with ACS exhibited significantly higher mean peak plasma sPLA2compared to subjects who never developed ACS (263±169 vs164±164 ng/mL respectively, p<0.001). The SCD-specific cutoff of sPLA2≥48 ng/mL yielded a sensitivity=85%, specificity=33%, NPV=89% and PPV=25% in predicting ACS. When comparing serial plasma sPLA2levels, mean sPLA2 at ED presentation was highest among patients presenting with ACS (198±182 ng/mL, n=18) with a positive chest x-ray (CXR) in the ED, followed by those with a negative CXR in ED who developed ACS during hospitalization (128±120 ng/mL, n=34). Notably, both groups demonstrated significantly higher sPLA2 levels at presentation compared to patients with No-ACS (106±125 ng/mL; p=0.02). This trend persisted throughout the hospital course, with mean sPLA2 levels continuing to rise by Day 2 and remaining significantly elevated at DC in both ACS groups compared to No-ACS (p<0.001). In the ACS cohort, 19 (36%) cases were classified as mild, 24 (46%) as moderate, and 9 (17%) were severe. Plasma sPLA2 levels at ED presentation increased with ACS severity, showing a stepwise rise from mild to moderate to severe cases. Subjects with severe ACS had a mean sPLA2 level that is 1.5-fold higher than those with mild disease (192±200 vs 122±140 ng/mL respectively) though not significant with the small sample size. A total of 16 patients presented to the ED with fever and 60 patients developed fever during their hospital stay (total n=76). Peak plasma sPLA2levels were significantly higher in febrile (n=76) versus afebrile (n=175) patients (254±19 vs 120±10 ng/mL, p<0.0001). ED plasma sPLA2levels correlated positively with several vital signs and clinical laboratory parameters including heart rate (r=0.42, p<0.001), respiratory rate (r=0.14, p=0.02), white blood cells (r= 0.48, p<0.001) and neutrophils (r=0.40, p<0.001). Moreover, sPLA2correlated negatively with lymphocytes (r=-0.42, p<0.001) and hemoglobin/hematocrit (r=-0.26, p<0.001). Conclusions: sPLA2 remains promising as a biomarker for ACS risk. We show it is also associated with ACS severity in children with SCD, supporting its potential role in acute clinical decision-making. Combined with other predictors, it may enhance early diagnosis and guide management.

  PublisherOA PDFDOI

• The ASH consa consortium on newborn screening in Africa for sickle cell disease: Interim updates of progress in screening, follow-up and supporting initiatives

  Blood · 2025-11-03

  articleOpen access

  Abstract Introduction: Children born with sickle cell disease (SCD) in sub-Saharan Africa (SSA) are at >50% risk for under age 5 mortality (U5M). To demonstrate whether newborn SCD screening in SSA and early entry into standard care will lower rates of U5M, the American Society of Hematology (ASH) established the Consortium on Newborn Screening in Africa (CONSA) in 10 sites in 7 countries: Ghana, Kenya, Liberia, Nigeria, Tanzania, Uganda and Zambia. The hypothesis is that early infant SCD screening and entry into standard continuous care will reduce U5M compared to historical estimates.Primary objectives are to determine: a) the population-based birth incidence of SCD; and b) effectiveness of early standardized care for preventing U5M consortium-wide at each country's site(s).Secondary objectives are to: a) establish SCD newborn screening programs and early intervention programs; and b) assess implementation and sustainability. Methods: CONSA is a multi-country health intervention program for centralized in-country newborn screening for SCD laboratory analysis of dried bloodspots, with enrollment of infants with SCD into early, continuous standardized care. Additional program needs have been identified, described here. Results: This report presents interim data on screening and clinical follow-up, highlighting several key initiatives developed to strengthen the CONSA program, which was launched in 2020. To date, results from 158,736 screened babies revealed 2201 (1.39%), with SCD, 25708 (16.20%) with S and 2531 (1.59%) C trait, and 1814 (1.14%) with another variant. Of those with screening results indicative of SCD, 693 underwent confirmatory testing, which established the diagnosis in 624 individuals (90%). To date, 847 (38.5%) have had an initial documented SCD clinical visit at a CONSA site. Others are receiving care at non-CONSA sites. Several initiatives are supporting CONSA, some through external resources:Screening and diagnostic capacity: Collaboration with a global technical source and in-country distributors has established networks to procure and distribute laboratory supplies, and provides technical support for laboratory guidance, training and troubleshooting to enhance laboratory capacity on isoelectric focusing using dried bloodspots and interpreting results.Sites were provided with supplies of the HemotypeSC device for rapid point-of-care diagnostic confirmation.Digital Health: A CONSA mobile phone app was created to support digital data collection with delayed cloud upload to the central database. Text messages are sent to families of infants with results of non-abnormal hemoglobin, reducing notification workload for data managers and nursing staff.Text messages are sent to affected families to remind them of follow-up visits.Health Education Initiative: Training with doctors, nurses, community health workers (CHWs), and other allied health professionals on SCD diagnosis and care has created networks of healthcare professionals for children living with SCD.More than 1,800 healthcare providers have been trained, with substantial pre- vs. post-test improvement.Improving clinical follow-up and retention in care: CONSA sites are partnering with local pediatric clinics to provide care, with referral to secondary or tertiary care as needed.The new CHW Initiative aims to support families of affected infants to increase documented visits for confirmatory diagnosis, enrollment and retention in care.Enhancing access to hydroxyureaSustainability: Measures to increase public awareness and reduce the stigma of SCD are underway to enhance in-country advocacy. CONSA country leaders are supported in developing key messages and strategies to engage local and national government leadership to promote their SCD efforts.Small grants are being provided through contracts with local community-based organizations to support community engagement and SCD advocacy at CONSA sitesSocial media outreach on SCD is being piloted to improve public awareness, specifically through promotional videos developed by musical artists collaborating with health education groups, distributed via popular global apps generally aimed at adolescents and young adults. Conclusions: Newborn screening encompasses a program beyond a test. Much progress has been made by the CONSA program that is tailored to local SSA strengths. The program is supported by several key initiatives to ensure effectiveness and sustainability.

  PublisherOA PDFDOI

• Sickle cell disease treatment with arginine therapy (STArT) – results of a phase-3 randomized controlled trial

  Blood · 2025-11-03 · 2 citations

  articleOpen access

  Abstract Background: Vaso-occlusive pain episodes (VOE) are the leading cause of emergency department (ED) visits and hospitalizations for patients with sickle cell disease (SCD). Yet, FDA-approved drugs for SCD-VOE are lacking. During SCD-VOE, patients develop an acute arginine (Arg) deficiency that is associated with adverse clinical outcomes including longer time-to-crisis-resolution (TCR), and greater total parenteral opioid use (TPO) during hospitalization. Multiple RCTs in the US, Brazil, Nigeria, and Egypt have established that supplementation with Arg is safe, has opioid-sparing effects, improves pain scores, blood pressure, and cardiopulmonary function and decreases length of hospital stay. We and others have shown that Arg increases nitric oxide production, improves mitochondrial function, and decreases both oxidative stress and biomarkers of hemolysis and inflammation. A phase-3 RCT was needed. Objective: To determine the efficacy and safety of intravenous (IV) Arg for SCD-VOE. Methods: We conducted a multicenter, double-blind, phase-3 RCT of IV Arg therapy in subjects aged 3-21 years with SCD-VOE receiving IV opioids at 10 US sites utilizing the Pediatric Emergency Care Applied Research Network (PECARN). Enrollment began in 2021 with a sample size goal of 360 patients. Within 12 hours of receiving their first dose of IV opioids, enrolled participants were randomized 1:1 to receive either 1) a one-time loading dose of IV Arg (200 mg/kg with a max of 20g) followed by a standard dose of 100 mg/kg (max 10g three times/day) or 2) placebo (normal saline) at equivalent volumes/duration as the study drug. Participants, research staff, and investigators were blinded to the patient's randomization. The primary outcome was TCR defined as the time in hours from study drug delivery to the last dose of IV opioid delivery. Secondary outcomes included TPO, defined as total IV morphine equivalents in mg/kg from study drug delivery to last parenteral opioid) and patient reported outcomes (PROMIS) at admission, discharge, and 7-12 days post-discharge. This protocol utilized FDA-IND#66943, registered with ClinicalTrials.gov (NCT04839354).Results: The STArT trial was halted early for futility to achieve the primary outcome of TCR. A total of 2,629 patients were screened, 1034 were eligible, 741 were approached, 293 consented, and 271 randomized who received study drug (129-Arg, 142-placebo) with enrollment milestones nearly a year ahead of schedule. Mean age ±standard deviation (SD) was 14±4 years, 51% were male, 71% had Hb-SS, 76% on hydroxyurea, and 41% had chronic pain (SCD-pain ≥15 days/month for the last 6 months). Demographics were similar across study arms. TCR was similar in the Arg arm vs placebo (82±80 vs. 89±152 hours, p=0.98). TPO was also similar across both arms (2.6±4.5 vs 1.9±2.8mg/kg, p=0.33). No differences in safety outcomes, adverse events, pain scores, PROMIS Pain Interference, Behavior or Fatigue were noted. 54 subjects had or developed acute chest syndrome (ACS). Among participants with ACS, there was a mean 76-hour difference in TCR in those treated with Arg vs placebo (108±98, n=29 vs. 184±327, n=25; p=0.16; median difference of 34 hours) that was not statistically significant. Larger than expected site variation, outliers and SD occurred; sample size recalculation based on STArT data would require randomization of over 900 subjects. Conclusion: All phase-3 RCTs for SCD-VOE to date have failed to meet their primary outcomes after promising phase-2 RCTs. The PECARN STArT trial is the latest addition to this list, stopped early for futility to achieve the primary outcome of TCR. There were no safety concerns. Ultimately, RCTs evaluating orphan drugs for SCD-VOE face significant challenges due to shorter hospital lengths of stay over the last 20 years, and extensive interpatient variability in TCR that could confound RCT outcome measures. Larger than expected SD and outliers occurred. Clinically relevant differences in TCR among patients with VOE-ACS treated with Arg similar to a prior RCT (Morris et al, Am J Hematol 2025) requires further study with a larger sample size. Dialogue between the FDA, ASH, SCD researchers and patients is needed to identify more ideal outcome measures beyond TCR, currently FDA-preferred for orphan drug approval.

  PublisherOA PDFDOI

• An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

  Transplantation and Cellular Therapy · 2025-02-01

  articleOpen access

  Lovo-cel, which consists of transplantation of autologous hematopoietic stem and progenitor cells encoding a functional anti-sickling β-globin gene, is approved for patients aged ≥12 y with SCD and a history of vaso-occlusive events (VOEs). We report updated outcomes for participants treated with lovo-cel from HGB-206 Group C (NCT02140554; phase 1/2) and HGB-210 (NCT04293185; phase 3) and examine how early pharmacodynamic (PD) parameters predict long-term clinical responses to lovo-cel. Data shown are from lovo-cel trial participants (HGB-206 completed and HGB-210 ongoing) treated as of Feb 2024; updated data will be presented. A post hoc, exploratory analysis utilized univariate logistic regression to estimate the association between post–lovo-cel PD parameters and clinical outcomes. A receiver operating characteristic curve determined the optimal HbA T87Q cutoff value, maximizing predictive power for complete resolution of VOEs (VOE-CR)/severe VOEs (sVOE-CR). As of Feb 2024, 55 participants (n=55; male, 61.8%; median [range] age, 21 [9-38] y) received lovo-cel per the current manufacturing process, including 14 participants <18 y. Median (range) time to last follow-up was 41.9 (0.9-72.3) months. Of 34 VOE-evaluable participants, 30 (88.2%) and 32 (94.1%) achieved VOE-CR and sVOE-CR in the 6-18 months post infusion. Among 46 evaluable participants, 41 (89.1%) achieved globin response (GR). Median (range) % peripheral blood (PB) HbA T87Q 6 months post infusion was 49% (26%-63%). Results were similar for adult (≥18 y) and pediatric (<18 y) subgroups. The lovo-cel safety profile reflects the known effects of underlying SCD and myeloablative conditioning . Analyses of HGB-206 (all groups) and HGB-210 data showed long-term PD and biologic responses can be predicted as early as 6 months post infusion (Fig. 1). PD parameters are highly predictive of clinical outcomes, including achieving GR and resolution of VOEs 6-18 months post infusion (Fig. 2). Increased HbA T87Q is associated with VOE-CR and sVOE-CR, with high confidence of achieving clinical outcomes above 33% PB HbA T87Q (Fig. 3). In HGB-206 Group C and HGB-210, the median HbA T87Q was 49% at ≥6 months. The HbA T87Q level is established by the drug product (DP), as PD response is significantly correlated with the DP % lentiviral vector (LVV)+ cells ( r =0.86, P <0.001). In participants from HGB-206 Group C and HGB-210, the median (range) % LVV+ cells is 83% (63%-93%). Among participants who are not VOE evaluable (<18 months follow-up; n=12), the median (range) % PB HbA T87Q is 53% (46%-59%) at 6 months. One-time lovo-cel treatment results in sustained HbA T87Q production and eliminated VOEs and sVOEs in a majority of participants. Models developed in this post hoc analysis enable prediction of the likelihood of VOE-CR and sVOE-CR using measurements as early as 6 months post lovo-cel infusion.

  PublisherDOI

• Post-Approval, Real-World Experience with Betibeglogene Autotemcel for Transfusion-Dependent Beta Thalassemia

  Transplantation and Cellular Therapy · 2025-02-01 · 1 citations

  article
  PublisherDOI

• Associations Between Pain Scores and Opioid Doses With Emergency Department Disposition and Return Visit Rates in Children With Sickle Cell Disease

  Pediatric Blood & Cancer · 2025-05-02 · 1 citations

  articleOpen access

  Rapid treatment and frequent reassessment of pain are key components of treatment guidelines for acute sickle cell disease (SCD) pain. Few studies, however, report the associations between emergency department (ED) pain scores, number of ED opioid doses, receipt of an opioid prescription, ED visit disposition, or ED return visits. This seven-site retrospective cohort study analyzed 4983 ED visits by children with SCD pain using electronic health record data from the Pediatric Emergency Care Applied Research Network Registry. ED pain scores included initial, last, and change in scores (initial minus last), measured on a 0-10 scale. Dispositions of discharge and hospital admission were included. Modified Poisson regression and the Cochran-Armitage test of trend were used for analysis. The median (IQR) initial pain score was 8.0 (6-10); last pain score was 5.0 (2-8); and median decrease was 2.0 (0-5). In multivariable analysis, last pain score was the best predictor of disposition. For the return visit analyses, of the 2377 visits discharged at index ED visit, 29% returned within 14 days. Higher initial and last ED pain scores were associated with increased return visits. Children with no opioid discharge prescription and ≥3 ED opioid doses had a return visit rate of 36% compared to 22% if the child received an opioid prescription and only one ED opioid. Increasing discharge opioid prescriptions and targeting interventions for those who receive multiple ED opioid doses could decrease return visits.

  PublisherOA PDFDOI

• Impact of HLA alloimmunization in gene-modified autologous stem cell transplant for transfusion-dependent thalassemia

  Blood · 2025-03-16 · 5 citations

  articleOpen access

  ABSTRACT: We report our single-center experience demonstrating that HLA class I alloimmunization predicts longer time to platelet engraftment, increased bleeding complications, and higher transfusion requirements in patients undergoing gene-modified hematopoietic stem cell transplant for transfusion-dependent β thalassemia.

  PublisherOA PDFDOI

• Revisiting acute chest syndrome (ACS) associated with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE): Insight from a prospective, multicenter Phase-3 randomized controlled trial

  Blood · 2025-11-03

  article

  Abstract Background: ACS occurs in up to 20% of hospitalized patients with SCD-VOE, often prolonging and complicating hospital stay. Despite its clinical significance, variations exist in the management and outcomes of ACS across institutions. Objective: Determine prevalence of ACS in children and young adults hospitalized for SCD-VOE and describe emergency department (ED) presentation, clinical course and practice variation across institutions. Methods: Cross-sectional analysis of data collected from a PECARN-endorsed multicenter, double-blinded, randomized, placebo-controlled phase-3 trial of intravenous arginine therapy in hospitalized patients with SCD-VOE aged 3-21 (NCT04839354) at 10 pediatric EDs across the US. ACS defined by radiology-interpreted chest radiograph (CXR) positive for new infiltrate and clinical team diagnosis. ACS severity was defined a priori as mild (no oxygen (O2) use or RBC transfusion), moderate (O2 use or transfusion), or severe (bilevel positive airway pressure (BiPAP) use, intubation, or pediatric intensive care unit (PICU) transfer). Results: 271 patients enrolled (median age 15[11,18] years, 51% male; 74% HbSS/Sb°; 76% on Hydroxyurea (HU). ACS occurred in 20% (n=54; median age 13[9,16] years; 76% male; 89% HbSS/Sb°; 80% on HU); 18 diagnosed in the ED, and 36 diagnosed during their hospitalization. 72% of patients with inpatient-diagnosed ACS had a negative CXR in the ED with a mean time to diagnosis of 2.4±1.6 days. Patients with ACS at any time were significantly younger (13[9,16] vs 15[12,18] years, p=0.004), predominantly male (76% vs 45%, p&amp;lt;0.001), and had HbSS/Sb° (89% vs 70%, p=0.0005) vs no ACS. In the ED, ACS patients had more O2 desaturations at &amp;lt;94% (37% vs 12%, p&amp;lt;0.001), higher frequency of cough (32%vs18%, p=0.04), wheeze (15%vs6%, p=0.04), and chest pain (59%vs39%, p=0.009) vs no ACS. No significant difference in fever across groups. 83% of patients with ACS presented with a normal respiratory exam in the ED. ACS patients had lower hemoglobin (g/dL;p&amp;lt;0.001) and lymphocyte counts (cells/µL;p=0.02), but higher %reticulocyte (p&amp;lt;0.001), white blood cell counts (x109/L;p=0.001), and absolute neutrophil counts (cells/µL;p=0.02) vs no ACS. Clinical outcomes were worse in patients with ACS vs no ACS, with longer length of stay (LOS; 139[93,189] vs 71[46,113] hours, p&amp;lt;0.001), higher opioid utilization (2.3[0.8,3.9] vs 1.1[0.4,2.1] mg/kg, p=0.001), more transfusions (61%vs14%, p&amp;lt;0.001), O2 use (76%vs20%, p&amp;lt;0.001), BiPAP use (20%vs2%, p&amp;lt;0.001) and PICU transfers (15%vs1%, p&amp;lt;0.001). Patients with inpatient-diagnosed ACS had longer LOS vs patients with ED-diagnosed ACS (142[113,213] vs 105[52,161] hours, p=0.03). 56% of ED-diagnosed ACS was mild, with no significant difference in LOS based on severity. However, only 25% of inpatient-diagnosed ACS were mild; LOS was significantly longer in moderate/severe vs mild ACS. Viral testing was performed in 67% of ACS patients, with 36% positivity for various viral pathogens without a predominant viral isolate. Across all sites, 96% of patients received antibiotics (50% ceftriaxone, 28% ampicillin-sulbactam, 7% vancomycin, 11% other) and 82% received azithromycin. Site-level use ranged from 0-100% for ceftriaxone and 50-100% for azithromycin. 80% received albuterol, 40% received inhaled steroids, and 11% received anticoagulation. No pulmonary emboli were reported. Of the 61% of patients transfused across sites, inter-site transfusion rate varied between 0-100%. Sex, age, ED O2 saturation &amp;lt;94%, hemoglobin, &amp; chest pain were independently associated with ACS in the multivariate model with an AUC=0.80. Conclusion: ACS remains common in patients with SCD-VOE (20%), with 14% of ACS diagnosed during their hospital stay. Two-thirds of subjects who developed ACS had a CXR done in the ED that was negative. ACS may have been missed in patients who did not get a CXR, and radiographic changes often lag behind symptoms. Lack of ACS-specific clinical signs and symptoms, including a normal ED respiratory exam in most patients, may delay diagnosis. Patients with inpatient-diagnosed ACS had a more severe hospital course vs those with ED-diagnosed ACS. Considerable practice variation exists in ACS management across institutions, with variable use of antibiotics and transfusion protocols. Further research on standardizing ACS treatment may be warranted. Oxygen desaturation in the ED was associated with a &amp;gt;3.6-fold greater risk of ACS, not included in prior ACS risk models.

  PublisherDOI

• Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment

  JAMA · 2025-10-22

  articleOpen access

  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%]; P = .002), which crossed the monitoring boundary for efficacy. Overall, there was no between-group difference in the number and type of adverse events. 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. Trial Registration: ClinicalTrials.gov Identifier: NCT03939637.

  PublisherOA PDFDOI

• Parent and adolescent perspectives on decision-making involvement: A qualitative comparison in sickle cell and diabetes clinics

  Patient Education and Counseling · 2025-10-24

  article
  PublisherDOI

Recent grants

• NIH Grant R55CA075485

  NIH · $100k · 1999

Frequent coauthors

• Janet L. Kwiatkowski

  258 shared

• Mark C. Walters

  University of California, San Francisco

  204 shared

• Suradej Hongeng

  Ramathibodi Hospital

  158 shared

• John B. Porter

  University College London

  148 shared

• Robert I. Liem

  Lurie Children's Hospital

  136 shared

• Ellis J. Neufeld

  St. Jude Children's Research Hospital

  128 shared

• Franco Locatelli

  Bambino Gesù Children's Hospital

  125 shared

• Patricia J. Giardina

  121 shared

Labs

• Thompson LabPI

Education

• B.A., Zoology

  Pomona College

  1979

• M.D.

  Tulane University School of Medicine

  1983

• Other, Health Services

  University of California, Los Angeles

  1987

Awards & honors

• Elias Schwartz MD Endowed Chair in Hematology, Children's Ho…