Doptelet (avatrombopag) / SOBI, Fosun Pharma - LARVOL DELTA

Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia (clinicaltrials.gov) - P2/3 | N=248 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

New P2/3 trial • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

The holistic trial: A comprehensive evaluation of two second-line therapeutic approaches for immune thrombocytopenia (ITP) – a pragmatic randomized controlled trial (ASH 2025) - P3 | "This trial aims to determine which of the two most widely used second-line therapies, rituximab or TPO-RA (avatrombopag) offers the better outcome and should be selected first, and whether specific patient subgroups may benefit more from one approach than the other. Furthermore, we seek to acquire knowledge on patient's perspectives, incorporating their preferences and experiences in treatment decisions, especially in relation to HR QoL . Funding statement: The study is sponsored by Østfold Hospital, Norway and supported by a grant from the Norwegian Regional Health Authority and SOBI."

Clinical • Hematological Disorders • Hepatitis B • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura

Trial in progress: Romiplostim N01 combined with immunosuppressive therapy in patients diagnosed with non-severe aplastic anemia, a Phase II study (ASH 2025) - P2 | "However, clinical data on romiplostim in NSAA remain limited, and no studies to date have evaluated the efficacy and safety of Romiplostim N01 in this population.Study Design and Methods We initiated a prospective, open-label, multi-centre, single-arm phase II trial (ChiCTR2500096280), conducted across multiple regions in China, to evaluate the efficacy and safety of Romiplostim N01 combined with cyclosporine or tacrolimus in patients diagnosed with NSAA and severe thrombocytopenia (platelet counts <30×10⁹/L)...Major eligibility criteria are: age ≥ 16, confirmed NSAA diagnosis, ECOG performance status of 0–2, QT interval <460 ms on electrocardiogram, adequate hepatic and renal function, prior TPO-RA recipients (including eltrombopag, hetrombopag, or avatrombopag) must complete a 1-month washout period before enrollment.Endpoints The primary endpoint is the overall hematologic response rate at weeks 12 and 24...A total of 40 subjects are..."

Clinical • P2 data • Anemia • Aplastic Anemia • Thrombocytopenia

A case for CD38 targeted therapy in multi-refractory immune thrombocytopenia (ASH 2025) - "Initial treatment with high-dose dexamethasone and IVIG was ineffective. He subsequently received rituximab, eltrombopag and avatrombopag...During weeks 8–14, he was treated with romiplostim, a second steroid pulse, mycophenolate, cyclophosphamide, and cyclosporine without response...While this study is ongoing, we present this case to highlight that daratumumab can be associated with a rapid platelet recovery in multi-refractory ITP, was safe and well tolerated with fostamatinib and danazol, and produced a durable response after a finite treatment course (12 weekly doses). In conclusion, for patients with prolonged, severe ITP unresponsive to conventional treatments, daratumumab offers a rational, mechanism-based intervention. Our case contributes to growing evidence supporting anti-CD38 immunotherapy in ITP and underscores the need for clinical trials to more broadly evaluate other plasma cell directed therapies for the treatment of ITP."

Clinical • Autoimmune Hemolytic Anemia • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Immune Thrombocytopenic Purpura • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • Thrombocytopenic Purpura

Avatrombopag: Beyond the technical sheet - a single-center experience (ASH 2025) - "Avatrombopag was initiated at 20 mg/day, increasing platelet counts to 40–90000/ml, enabling safe initiation of Dabigatran Etexilato. These encouraging observations underscore the need for prospective, controlled studies to validate the efficacy and safety of Avatrombopag in these off-label settings and to better define its role in personalized thrombocytopenia management. Ongoing real-world evidence is essential to inform the evolution of treatment guidelines"

Clinical • Atrial Fibrillation • Cardiovascular • Diabetes • Fibrosis • Hematological Disorders • Hemophilia • Hemophilia A • Hepatology • Hypertension • Immune Thrombocytopenic Purpura • Immunology • Metabolic Disorders • Portal Hypertension • Rare Diseases • Thrombocytopenia • Thrombocytopenic Purpura • Type 2 Diabetes Mellitus

Comparative effectiveness of second-line therapies for chronic immune thrombocytopenia: A network meta-analysis of RCTs (ASH 2025) - "The treatment groups were: Avatrombopag (96), Eltrombopag (283), Fostamatinib (101), Rilzabrutinib (133), Romiplostim (2116), and Rozanolixizumab (41)...Rituximab was analyzed indirectly and showed moderate efficacy... This NMA provides a comparative analysis of second-line therapies for chronic ITP, with Rozanolixizumab identified as the most effective treatment. Despite variability in safety profiles, all therapies demonstrated significant clinical benefits, supporting the adoption of personalized treatment strategies."

HEOR • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Avatrombopag: Beyond efficacy (ASH 2025) - "Prior to switch, 36.36% of patients received concomitant medication for ITP (preferably Prednisone in doses < 5-10 mg/day)...2, Cooper et al, The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK, K. J. Mark...Pascual C. et al, Avespa Study: Effectiveness and Safety of Avatrombopag in Immune Thrombocytopenia (ITP). a Real-World Study of the Spanish ITP Group (GEPTI).Blood (2024)144, Supplement 1, 713"

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

Patient-reported outcomes of avatrombopag for chronic immune thrombocytopenia: Interim analysis of the Phase 4 adopt study (ASH 2025) - "This interim analysis of the ADOPT study supports the real-world effectiveness and safety ofAVA for treating people with ITP. Improvements in PROs, including health status, HRQoL, and ITP-relatedactivity impairment, in addition to clinical outcomes, underscore the potential of AVA to benefit patientswith ITP."

Clinical • P4 data • Patient reported outcomes • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Mood Disorders • Psychiatry • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

TPO-ras for persistent thrombocytopenia following CAR-T therapy in multiple myeloma: A multicenter clinical experience (ASH 2025) - "Univariate andmultivariate analyses were conducted between patients with PT (cohort 1) and without PT (cohort 2).Patients with PT were treated with thrombopoietin receptor agonists (TPO-RAs), including: hetrombopag(initial dose: 2.5 mg/day), avatrombopag (initial dose: 20 mg/day), eltrombopag (initial dose: 50 mg/day).Response was defined as transfusion independency along with resolution of platelets > 50×10⁹/L forthree consecutive values on different days. Hyperferritinemia post-CAR-T independently predicted PT, while TPO-RAs demonstrated both platelet-boosting effects and marrow recovery. Further prospective studies are warranted to validate thesefindings and optimize therapeutic protocols."

Clinical • IO biomarker • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Thrombocytopenia

Real-world study on thrombopoietin receptor agonists combined with rituximab in the treatment of Relapsed/Refractory primary immune thrombocytopenia (ASH 2025) - "Combination regimens utilized avatrombopag (n=23), eltrombopag (n=32), or hetrombopag (n=33).Rituximab was administered as standard-dose (375 mg/m² weekly ×4; n=41) or reduced-dose regimens(n=47). Of 42 patients who discontinued TPO-RAs from thecombination regimen, cessation reasons included: drug-related hepatotoxicity (n=2), inadequatetherapeutic response (n=27), and 13 patients achieving sustained remission after complete treatmentwithdrawal. After combination therapy, only 4 cases (4.5%) experienced grade ≥3 adverse events (2 cases ofpulmonary infection and 2 cases of abnormal liver function).ConclusionThis multicenter study demonstrates that combination therapy with TPO-RAs and rituximab appears tobe a favorable option for relapsed/refractory ITP patients, including patients with prior TPO-RAs failure."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Real world data on the use of thrombopoietin receptor agonists in patients after allogeneic hematopoietic stem cell transplantation (ASH 2025) - "Eltrombopag was the most frequently used TPO-RA followed by Romiplostim andAvatrombopag in 20 (74%), 4 (15%) and 3 (11%) patients, respectively. Given the observed data we conclude that TPO-RA treatment after allogeneic HSCT might be avaluable option for patients with prolonged or secondary thrombocytopenia. However, prospectiveanalyses are necessary to determine a potential difference between TPO-RA effectiveness and plateletrise in the course of delayed graft function."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Gastroenterology • Graft versus Host Disease • Hematological Disorders • Immunology • Thrombocytopenia • Transplantation • CD34

Prolonged thrombocytopenia following diverse CAR-T cell therapies: High incidence, inflammatory risk factors, and a comparative efficacy analysis of TPO-receptor agonist strategies in a real-world cohort (ASH 2025) - "The efficacy of TPO-RA interventions—includingmonotherapy (Eltrombopag, Avatrombopag, Hetrombopag, Lusutrombopag, Romiplostim, or rhTPO) andcombination strategies—was evaluated. Prolonged, severe thrombocytopenia is a frequent, inflammation-driven complication of CAR-T therapy. Our real-world data demonstrate a clear efficacy hierarchy among TPO-RAs, with romiplostimbeing the most effective single agent. Crucially, combination TPO-RA strategies, particularly thoseinvolving romiplostim or sequential rhTPO, significantly accelerate platelet recovery in severe caseswithout increasing toxicity."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • B Cell Non-Hodgkin Lymphoma • Cardiovascular • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • IFNG

Mini-ATG/TBI conditioning regimen facilitates cure of acquired aplastic anemia in children via unrelated umbilical cord blood transplantation (ASH 2025) - P4 | "The mini-ATG/TBI regimen comprised: rabbit ATG (rATG, 2.5 mg/kg)on day -7, total body irradiation (TBI, 3 Gy) on day -7, fludarabine (FLU, 40 mg/m²) on days -6 to -2, andcyclophosphamide (CTX, 40 mg/m²) on days -4 and -2...In the mini-ATG/TBI group,odd-numbered patients received avatrombopag starting on day +7 to promote platelet engraftment,while even-numbered patients received thrombopoietin (TPO)... These data support the mini-ATG/TBI regimen as an effective conditioning approach forUCBT in acquired aplastic anemia, demonstrating particular utility in pediatric patients or low bodyweight adults.Key words: Aplastic Anemia; Unrelated Umbilical Cord Blood Transplantation; mini ATG"

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Septic Shock • Transplantation

Clinical characteristics, treatment strategies, and response patterns in evans syndrome: Initial analysis from the spanish resti cohort (ASH 2025) - "Cyclosporine was used in two patients with dual-lineage cytopenias,resulting in AIHA response in 41 days and neutrophil recovery in 15 days.Fostamatinib was used in four patients with concurrent ITP and AIHA...The median time from diagnosis to fostamatinib initiation was 531 days.All patients were on concomitant therapy (corticosteroids or avatrombopag)...This analysis from RESTI highlights the clinical heterogeneity and treatment challenges in Evanssyndrome. Corticosteroids remain first line of therapy but theyt are associated with incomplete ortransient responses in many patients. Rituximab demonstrated moderate efficacy across lineages, whilefostamatinib showed rapid, durable responses in both ITP and AIHA without observed toxicity, evenallowing treatment withdrawal in some cases."

Clinical • Autoimmune Hemolytic Anemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Neutropenia • Thrombocytopenia • Thrombocytopenic Purpura

Real-world safety and efficacy of avatrombopag in adults with immune thrombocytopenia: A systematic review and meta-analysis. (ASH 2025) - "In addition, the analysis confirms high rates of both complete andpartial platelet response, along with favourable data on time to response, treatment discontinuation,and AE. Overall, these results reinforce the real-world safety and efficacy of AVA."

Real-world • Real-world evidence • Retrospective data • Review • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Switching ITP patients to avatrombopag from other TPO-ras: A real-world experience of the spanish ITP group (GEPTI). (ASH 2025) - "Nor has the ability of AVA to restorePCs in ITP patients who did not respond to eltrombopag or romiplostim been addressed. Six treatment-unrelated deaths were documented during the study.Collectively, these results support the notion that AVA is a safe and effective tool to use instead of otherTPO-RAs to treat ITP, regardless of whether switching is due to patient preference, poor tolerability orinability to restore PCs. The successful use of low doses to maintain response in the long term invites usto consider AVA as a primary option when TPO-RA-based treatment of ITP is scheduled."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Myelofibrosis • Thrombocytopenia • Thrombocytopenic Purpura • Thrombocytosis

Real-world treatment patterns & outcomes among patients with immune thrombocytopenia (ITP) who switched treatment from eltrombopag or romiplostim to avatrombopag in the United States: Results from the real-AVA 3.5 study (ASH 2025) - "In this RW study evaluating the effectiveness of AVA treatment following a switch from ELT or ROMI, mostpatients achieved or maintained a clinically meaningful and durable platelet response throughout thestudy observation period. The need for rescue therapy was rare and all patients on concomitant steroidswere able to discontinue them, further supporting the effectiveness of AVA in patients with primary ITPwho switched from ELT or ROMI."

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

Avatrombopag improves platelet engraftment after haploidentical hematopoietic stem cell transplantation: A randomized, double-blind, multicenter, placebo-controlled trial (ASH 2025) - "Overall, this represents the first randomized, double-blind, multicenter, placebo-controlled trial toconfirm that avatrompag is effective and safe in improving platelet recovery after haplo-HSCT."

Clinical • Bone Marrow Transplantation • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Liver Failure • Thrombocytopenia • Thrombosis • Transplantation

Immunotherapy-Associated Immune Thrombocytopenia: Treatment Paradigms (ASH 2023) - "We categorized patients into two cohorts: IO-ITP, those with a diagnosis of ITP after IO use (pembrolizumab, nivolumab, atezolizumab, avelumab, durvalumab, ipilimumab) and a diagnosis of neoplasm (ICD-10: C00-D49); and p-ITP: those with ITP without neoplasm nor IO use...We investigated the use of first-line and rescue therapies: glucocorticoids and IVIG; second-line therapies: thrombopoietin agonists (TPO-RAs: romiplostim and eltrombopag) and rituximab; third-line therapies: cyclophosphamide, azathioprine, cyclosporine, mycophenolate mofetil, bortezomib, avatrombopag and fostamatinib; and splenectomy, based on guidelines for management of p-ITP... Our study describes treatment patterns in IO-ITP, an uncommon but challenging disease. We found that individuals with IO-ITP are generally younger and predominantly male as compared to p-ITP, highlighting different patient profiles. We further demonstrated some distinctions in the management of these two conditions, possibly..."

Hematological Disorders • Immune Thrombocytopenic Purpura • Oncology • Skin Cancer • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and Safety of Avatrombopag on Thrombocytopenia after Chemotherapy in Acute Leukaemia (ASH 2023) - "Chemotherapy regiments for patients with AML included decitabine combined with venetoclax (n=5), decitabine combined with arabinoside (n=3), arsenic trioxide combined with retinoic acid (n=3), azacytidine combined with venetoclax (n=2), azacytidine combined with medium-dose arabinoside (n=1). Chemotherapy regiments for patients with ALL included CD19 specific CAR-T cells (n=3), decitabine (n=2), blinatumomab (n=2), IVP (idarubicin, vindesine, and prednisone) (n=1), azacytidine combined with venetoclax and chidamide (n=1)... Avatrombopag is effective on increasing platelet counts in patients with acute leukaemia after chemotherapy, with a good safety profile. It is a suitable therapeutic option for thrombocytopenia after chemotherapy."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Hepatology • Immune Thrombocytopenic Purpura • Leukemia • Oncology • Thrombocytopenia • Thrombocytopenic Purpura

Combination of anti-CD20 and hetrombopag in relapsed/refractory immune thrombocytopenia: a case series. (PubMed, Front Med (Lausanne)) - "Accordingly, the patient was treated with avatrombopag monotherapy, which maintained the PLT count at normal levels...After treatment with ripertamab-hetrombopag, the patient received combination therapy with hetrombopag, glucocorticoids, tacrolimus, and hydroxychloroquine, with the PLT count being maintained within the normal range. The other two patients remained in sustained CR throughout the follow-up period. Combining anti-CD20 monoclonal antibody with hetrombopag may offer therapeutic benefits in patients with relapsed/refractory ITP."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025 (PRNewswire) - "Sobi's innovative portfolio including data from efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib....Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria; Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report; User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria."

Clinical data • Aplastic Anemia • Chronic Myelomonocytic Leukemia • Hemophagocytic lymphohistiocytosis • Hemophilia A • Immune Thrombocytopenic Purpura • Myelofibrosis • Paroxysmal Nocturnal Hemoglobinuria

Avatrombopag Safe and Effective in Treatment of Severe Refractory Thrombocytopenia in a Pediatric Patient with Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia Following Allogeneic Hematopoietic Stem Cell Transplantation (ASH 2023) - "Case Presentation: We present a 14-year-old Hispanic male with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who received a maternal haploidentical HSCT with myeloablative conditioning and post-transplant cyclophosphamide...Multiple treatments including romiplostim; eltrombopag; high dose immune globulin (IVIG); rituximab; methylprednisolone; and decitabine were not successful in improving platelet count (Figure 1)...Patient continues to maintain platelets above 100 x10 3/uL with no medication toxicities or adverse events noted. Patient has been able to return to his pre-alloHSCT lifestyle including contact sports and re-immunizations."

Clinical • Acute Lymphocytic Leukemia • Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Leukemia • Neutropenia • Oncology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation • CXCL9

Clinical and Economic Burden of Illness in Patients with Persistent or Chronic Primary Immune Thrombocytopenia Treated with TPO-Ras and Rituximab (ASH 2023) - "Patients with ITP lasting for ≥3 months (persistent or chronic primary ITP), and who do not respond adequately or maintain a response to initial therapy, are usually treated with advanced therapies such as thrombopoietin receptor agonists (TPO-RAs [avatrombopag/romiplostim/eltrombopag]), rituximab, and fostamatinib. All-cause hospitalization was high in this population. Further studies are needed to understand long-term clinical and economic outcomes among ITP patients."

Clinical • HEOR • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Diabetes • Gastroenterology • Heart Failure • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Metabolic Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Struck By Tsunami: Difficult Case of a Severe Refractory ITP (ASH 2024) - "While the majority of cases present with mild to moderate thrombocytopenia, severe cases can pose significant challenges in diagnosis and management.We present the case of a 65-year-old male with severe ITP refractory to initial standard treatment including steroids and intravenous immunoglobulin (IVIG) requiring further management including rituximab, romiplostim, Fostamatinib, romiplostim, Efgartigimoid alfa-fcab , eltrombopag and partial splenic embolization.Case presentationOur patient is a 65-year-old male with a significant medical history, including end-stage renal disease (ESRD), diffuse large B-cell lymphoma (DLBCL) status post-stem cell transplant in 2015, and chronic immune thrombocytopenic purpura (ITP)...Also during hospital stay General Surgery were consulted to assess the patient for splenectomy, which was deferred due to low platelet levels and to allow for further evaluation of the effects of embolization.Following two weeks from the second partial..."

Clinical • B Cell Lymphoma • Cardiovascular • Chronic Kidney Disease • Diffuse Large B Cell Lymphoma • Gastroenterology • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Lymphoma • Nephrology • Non-Hodgkin’s Lymphoma • Oncology • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis