Doptelet (avatrombopag) / SOBI - LARVOL DELTA

Letter to the editor, regarding "Stroke, idiopathic thrombocytopenic purpura and avatrombopag: two cases of coincident events" recently published by Boy and colleagues. (PubMed, Neurol Sci) - No abstract available

Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Ischemic stroke • Thrombocytopenia • Thrombocytopenic Purpura

Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study. (PubMed, Lancet Haematol) - P3 | "Avatrombopag is an effective oral treatment for children and adolescents with ITP for at least 6 months and has a reassuring safety profile in the paediatric population. Avatrombopag could provide an important treatment option for paediatric ITP."

Journal • P3 data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Pain • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

A phase 3 study of the efficacy and safety of avatrombopag in Japanese adults with chronic immune thrombocytopenia. (PubMed, Int J Hematol) - "Avatrombopag demonstrated a rapid and durable platelet response and was well tolerated in Japanese patients with chronic ITP. Long-term safety and efficacy are being assessed in the ongoing extension phase."

Journal • P3 data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Drugs and patient's dissatisfaction reporting: From a disproportionality pharmacovigilance analysis to social pharmacology. (PubMed, Therapie) - "Despite its mandatory limitations (underreporting, selective reporting…), this study shows that pharmacovigilance data could help to understand some aspects of social pharmacology, here, patient dissatisfaction with their drug treatment."

Adverse events • Journal • Dermatology

Patterns and Predictors of Response to Thrombopoietin Receptor Agonists in Children With ITP (ASPHO 2025) - "NR rates to eltrombopag, romiplostim and avatrombopag were 19%, 26% and 20%, respectively...All tried rituximab: 2 achieved response... Rates of NR remain similar across TPO-RAs. Patients with secondary ITP, especially SLE, can achieve sustained responses to TPO-RAs. Younger age at diagnosis was associated with NR to TPO-RAs."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Inflammatory Arthritis • Lupus • Pediatrics • Systemic Lupus Erythematosus • Thrombocytopenia • Thrombocytopenic Purpura

Eltrombopag-Induced Iron Deficiency Anemia in Chronic ITP (ASPHO 2025) - " Case 1: A 4-year-old female diagnosed with ITP experienced transient responses to initial therapies (IVIG, corticosteroids, rituximab). Eltrombopag is effective in increasing platelet counts in pediatric chronic ITP. However, its chelating properties pose a risk of persistent IDA, necessitating vigilant iron monitoring and supplementation. Transitioning to alternative thrombopoietin receptor agonists, such as Romiplostim or Avatrombopag, may help mitigate this complication."

Anemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • DMRT1

BASELINE CORRELATES WITH DURABILITY OF AVATROMBOPAG RESPONSE: A PHASE 3B TRIAL FOR TREATMENT OF CHILDREN WITH ITP (EHA 2025) - P3 | "Despite a high proportion of prior TPO-RA failure, durable platelet response was noted across a variety of clinical and demographic characteristics. Given the small sub-groups numbers, some characteristics may yield higher response rates and should be further studied."

Clinical • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

CONSISTENT RESPONSE TO AVATROMBOPAG ACROSS VARIOUS BASELINE CHARACTERISTICS: RESULTS FROM THE PHASE 3-B TRIAL FOR THE TREATMENT OF CHILDREN WITH IMMUNE THROMBOCYTOPENIA (EHA 2025) - P3 | "A consistent platelet response to AVA was noted across a variety of baseline characteristics. High AVA response rates were noted despite 77% of patients receiving multiple TPO-RAs prior to study entry and 73% failing to respond to prior TPO-RAs."

Clinical • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

EFFICACY OF SWITCHING TREATMENT OF AVATROMBOPAG FOLLOWING FAILURE WITH PREVIOUS THROMBOPOIETIC AGENTS IN THROMBOCYTOPENIA PATIENTS (EHA 2025) - "Previous thrombopoietic therapy was as follows, 59.26% (16/27) had used hetrombopag, 18.52% (5/27) had used eltrombopag, 3.70% (1/27) had used romiplostim, and 18.52%(5/27) had combination treatments.. Our data shows that switching to AVA is an effective treatment for ITP and CTIT."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Oncology • Pain • Thrombocytopenia • Thrombocytopenic Purpura

INDIRECT COMPARISON IN REAL LIFE BETWEEN 2 ORAL TREATMENTS IN ITP USING ARTIFICIAL INTELLIGENCE (EHA 2025) - "The potential of ChatGPT4o, in medicine, not only in hematology (https://codigorojo.tech/home/) is many and varied, as Van de Wyngaert (Haemophilia (2023) doi10.1111/hae.14858) reminds us, stating that "ChatGPT4o can be a valuable tool for patients and healthcare professionals, but it should not replace medical consultations or the experience of a multidisciplinary hemophilia team" Aims: Make an indirect comparison in terms of safety and efficacy between fostamatinib and avatrombopag base don results of two national studies in real life (Table 1). Obviously, this analyses have limitations; in general, statistical analysis performed is simplified and lacks adequate adjustments. Although the simplified analysis can provide a global view of the efficiency of both drugs, we must consider the mentioned limitations for an adequate interpretation of results. This analysis could be indicative for choosing between one or the other drug, considering the patient's..."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis

REAL WORLD EXPERIENCE OF DISCONTINUATION OF THROMBOPOIETIN RECEPTOR AGONIST (TPO-RA) IN PRIMARY IMMUNE THROMBOCYTOPENIA (ITP) (EHA 2025) - "The combinations were avatrombopag + fostamatinib; eltrombopag + mycophenolate; romiplostim + mycophenolate; romiplostim + mycophenolate + fostamatinib.29 patients were eligible to stop their TPO-RA. TPO-RA therapy can be safely discontinued in selected patients with primary ITP. The majority of patients stopping TPO-RA sustained adequate platelet counts to maintain haemostasis (≥20-30 x 109/L). A trial of TPO discontinuation could be considered in more patients in our centre, but shared-decision making is important in patients where there is clear reason for continued TPO-RA therapy."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

ENHANCING OUTCOMES IN HEPATITIS-ASSOCIATED APLASTIC ANEMIA: THE ROLE OF THROMBOPOIETIN RECEPTOR AGONISTS AS AN ADJUNCT TO STANDARD IMMUNOSUPPRESSIVE THERAPY (EHA 2025) - "The standard management strategies for HAAA primarily consist of immunosuppressive therapy (IST), typically involving antithymocyte globulin (ATG) combined with cyclosporine (CsA), as well as allogeneic hematopoietic stem cell transplantation (allo-HSCT) for eligible patients...The ORR of patients treated with avatrombopag, eltrombopag and hetrombopag were 37.5% vs 40% vs 100%, and 62.5% vs 60% vs 100% at 3, 6 months after IST, respectively... In conclusion, the addition of TPO-RAs to standard IST demonstrated a favorable safety profile and was associated with a significantly higher ORR in patients with HAAA."

Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hepatology • Inflammation • Liver Failure

EFFECTS OF THROMBOPOIETIN AND THROMBOPOIETIN RECEPTOR AGONISTS ON PLATELET ACTIVATION AND THROMBOSIS (EHA 2025) - "TPO and TPO-RA, including romiplostim, eltrombopag, hetrombopag and avatrombopag, can enhance platelet activation and thrombosis in mice. However, these agents do not increase the risk of complete vascular occlusion after following arterial injury."

Cardiovascular • Hematological Disorders • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Thrombosis

REAL-WORLD TREATMENT PATTERNS & CLINICAL OUTCOMES IN THROMBOPOIETIN RECEPTOR AGONIST NAIVE PATIENTS WITH IMMUNE THROMBOCYTOPENIA TREATED WITH AVATROMBOPAG: INTERIM RESULTS FROM THE REAL-AVA 3.0 STUDY (EHA 2025) - "In this RW study evaluating the effectiveness of AVA as a first-line TPO-RA in primary ITP, interim results demonstrate a high response rate in adult patients with low PCs at AVA initiation, which was maintained throughout the majority of the follow-up period. All patients on concomitant medications were able to reduce or discontinue them, and few patients required rescue treatment while receiving AVA. Additional longitudinal follow-up is warranted to better understand the long-term durability of AVA."

Clinical • Clinical data • HEOR • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

EFFECTIVENESS AND SAFETY OF AVATROMBOPAG FOR TREATMENT OF IMMUNE THROMBOCYTOPENIA IN OLDER PATIENTS AND THOSE WITH COMORBIDITIES OR PRIOR TPO-RA EXPOSURE: INTERIM RESULTS FROM THE PHASE 4 ADOPT STUDY (EHA 2025) - "In total, the patient distribution across groups was ≥65 y, n=73; comorbidities with increased TEE risk, n=89; and prior TPO-RA exposure, n=128 (eltrombopag only [ELT], n=58; romiplostim only [ROM], n=45; ELT+ROM, n=25) (Table). In this interim analysis of real-world data from ADOPT, AVA was effective across patients aged ≥65 y and those with comorbidities or prior TPO-RA exposure, with no new safety concerns identified. Understanding AVA's profile among these subgroups will provide valuable insights for optimizing treatment strategies in diverse real-world populations."

Clinical • P4 data • Cardiovascular • Chronic Kidney Disease • Genetic Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Nephrology • Obesity • Oncology • Pulmonary Embolism • Renal Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

RESPONSE TO AVATROMBOPAG AMONG PATIENTS WITH CHRONIC AND ACUTE/PERSISTENT PRIMARY IMMUNE THROMBOCYTOPENIA: THE REAL-AVA 2.0 REAL-WORLD STUDY (EHA 2025) - "The majority of patients with acute/persistent or chronic ITP achieved treatment response across all PC thresholds, with a median time to response as early as 14 days. These findings suggest that treatment response to AVA was consistent regardless of ITP disease duration before initiating AVA.REAL-AVA 2.0 was funded by Sobi, Inc."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

EFFECTIVENESS AND SAFETY OF AVATROMBOPAG FOR THE TREATMENT OF ADULTS WITH NEWLY DIAGNOSED, PERSISTENT, OR CHRONIC IMMUNE THROMBOCYTOPENIA: INTERIM RESULTS FROM THE PHASE 4 ADOPT STUDY (EHA 2025) - "In this interim analysis of real-world data from the ADOPT study, avatrombopag was shown to be effective among adult patients with newly diagnosed, persistent, and chronic ITP treated in clinical practice, suggesting that the benefits of avatrombopag treatment among patients with ITP are similar across disease phases. No new safety concerns have been identified to date."

Clinical • P4 data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Ischemic stroke • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

EVALUATION OF EFFICACY AND SAFETY OF AVATROMBOPAG IN CHILDREN WITH IMMUNE THROMBOCYTOPENIA BASED ON DISEASE DURATION: RESULTS FROM THE AVATROMBOPAG PHASE 3-B PEDIATRIC TRIAL (EHA 2025) - P3 | "AVA is an effective, durable, and safe therapy in children with ITP, regardless of disease duration."

Clinical • P3 data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

EFFICACY AND SAFETY OF AVATROMBOPAG FOR THE TREATMENT OF PEDIATRIC IMMUNE THROMBOCYTOPENIA IN THE OPEN-LABEL EXTENSION OF A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL (EHA 2025) - "Avatrombopag demonstrated sustained efficacy during this OLE, with a cumulative safety profile consistent with that of the core phase and no new or unexpected safety concerns identified. These findings suggest the long-term efficacy and safety of avatrombopag for treating children with ITP."

Clinical • P3 data • Anemia • Aplastic Anemia • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Thrombocytosis • Thrombosis

PLATELET RESPONSE TO AVATROMBOPAG AMONG PATIENTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA WHO SWITCHED FROM ELTROMBOPAG OR ROMIPLOSTIM: THE REAL-AVA 2.0 REAL-WORLD STUDY (EHA 2025) - "The majority of patients who switched to AVA from another TPO-RA for the treatment of primary ITP achieved or maintained PC response with AVA. This suggests consistency of response to AVA treatment, regardless of the prior TPO-RA or the reason for treatment switch.REAL-AVA 2.0 was funded by Sobi, Inc."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

DURABILITY OF RESPONSE TO AVATROMBOPAG AMONG PATIENTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA: THE REAL-AVA 2.0 REAL-WORLD STUDY (EHA 2025) - "Patients who initiated AVA for the treatment of primary ITP were able to maintain response for over 10 months with robust durability of response across various response thresholds, including a median durability of 95.9% at PC≥30k/µL, 93.4% at PC ≥50k/µL and 76.5% at PC ≥100k/µL, indicating a stable, long-term response to AVA.REAL-AVA 2.0 was funded by Sobi, Inc."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

CLINICALLY MEANINGFUL RESPONSE TO AVATROMBOPAG: A PHASE 3B TRIAL FOR TREATMENT OF CHILDREN WITH ITP (EHA 2025) - P3 | "A significant and rapid response to AVA was seen in this heavily pretreated pediatric ITP population, regardless of how the response was measured."

Clinical • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

EFFICACY AND SAFETY OF AVATROMBOPAG IN RELAPSED OR REFRACTORY SEVERE APLASTIC ANAEMIA: RESULTS OF THE DIAAMOND-AVA-NEXT TRIAL (EHA 2025) - P2 | "Participants may have been on ciclosporin and were permitted to have co-administration of equine or rabbit ATG if assessed as clinically indicated by the treating clinician. Avatrombopag in addition to standard of care in relapsed or refractory sAA patients resulted in OR of 50% and ACE of 7% at 6 months."

Clinical • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Septic Shock

Clinically Meaningful Response to Avatrombopag for Treatment of Pediatric Immune Thrombocytopenia (ASPHO 2025) - P3 | "A significant and rapid response to AVA was seen in this heavily pretreated pediatric ITP population, regardless of how the response was measured."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

Baseline Correlates With Durability of Avatrombopag Response in Pediatric Immune Thrombocytopenia (ASPHO 2025) - P3 | "Despite a high proportion of prior TPO-RA failure, durable platelet response was noted across a variety of clinical and demographic characteristics. Given the small sub-groups numbers, some characteristics may yield higher response rates and should be further studied."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura