Ayvakit (avapritinib) / CStone Pharma, Sanofi - LARVOL DELTA

Refractory Ascites in Systemic Mastocytosis Mimicking Decompensated Cirrhosis (ACG 2026) - "Diuretics were discontinued, and avapritinib improved abdominal distension and functional status...Recognizing this is clinically important, as diuretic therapy may provide limited benefit, whereas targeted therapy directed at the underlying mast cell disorder may improve outcomes Figure: Sagittal CT abdomen/pelvis demonstrating large-volume ascites with marked splenomegaly and diffuse abdominal lymphadenopathy. Figure: Liver biopsy demonstrating preserved hepatic architecture with mild portal-based inflammatory infiltrate and focal portal fibrous expansion without bridging fibrosis or cirrhosis."

Cardiovascular • Fibrosis • Gastric Cancer • Gastrointestinal Cancer • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Portal Hypertension

When the Biopsy Says No but the Tumor Says Yes: Metastatic GIST With PDGFRA M844 Deletion (ACG 2026) - "The D842V substitution, the most common PDGFRA exon 18 variant, confers primary resistance to imatinib and is the best-characterized target of avapritinib. Third, avapritinib produced a meaningful radiographic response in this rare genotype, adding real-world evidence to the sparse literature on avapritinib efficacy beyond D842V. These findings support comprehensive molecular profiling in GIST and the need for prospective registries to characterize TKI responsiveness in uncommon PDGFRA variants."

Biopsy • Metastases • Alopecia • Fibrosis • Gastrointestinal Stromal Tumor • Hepatology • Immunology • Oncology • Sarcoma • PDGFRA

Progression-Free Survival With Second, Third, and Fourth Line Tyrosine Kinase Inhibitors After Imatinib Failure in Gastrointestinal Stromal Tumors: A Genetic Mutation-Stratified Systematic Review and Meta-Analysis (ACG 2026) - "Masitinib did not improve PFS versus sunitinib. In the VOYAGER trial, avapritinib did not improve progression-free survival compared with regorafenib in the overall study population, but it showed activity in patients with PDGFRA D842V mutations... 11 randomized trials including 2,181 patients with advanced gastrointestinal stromal tumors after imatinib failure were included. Overall, active TKIs significantly improved progression-free survival (PFS) compared with placebo or best supportive care (HR 0.32, 95% CI 0.21â0.51) although heterogeneity was substantial. Sunitinib, regorafenib, ripretinib, pazopanib, and imatinib rechallenge showed benefit in delaying disease progression compared with placebo or best supportive care, whereas Nilotinib did not with best supportive care, with or without imatinib or sunitinib."

Retrospective data • Review • Stroma • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

Efficacy of ripretinib in advanced gastrointestinal stromal tumor and treatment options after resistance: a retrospective real-world study​ (PubMed, Zhonghua Wei Chang Wai Ke Za Zhi) - "After disease progression (PD), the following treatment strategies could be selected after multi-disciplinary team (MDT) (1) the dose of ripretinib was increased from 150 mg once daily to 150 mg twice daily (ripretinib dose-escalation group); (2) The standard-dose ripretinib (150 mg, once daily) was combined with local treatment, including surgery, interventional therapy and radiotherapy (ripretinib combined with local treatment group); (3) standard-dose ripretinib combined with frontline TKI, including imatinib, sunitinib and regorafenib (ripretinib combined with frontline TKI group); (4) other TKI treatment, including imatinib, sunitinib, regorafenib and avapritinib (other TKI treatment group). Ripretinib can serve as an effective treatment option for Chinese patients with advanced GIST after failure of first-line or multiple prior TKI therapies, and it demonstrates a favorable safety profile. Although no standard treatment is currently available for patients with..."

Journal • Real-world evidence • Retrospective data • Cardiovascular • CNS Disorders • Gastrointestinal Stromal Tumor • Hepatology • Oncology • Renal Disease • Sarcoma • Vascular Neurology

Clonal Architecture, Spontaneous Tumor Lysis Syndrome, and Sequenced KIT Inhibition in Systemic Mastocytosis With Associated Hematologic Neoplasm Involving Atypical Chronic Myeloid Leukemia: A Case-Based Analysis of Three Unresolved Management Challenges (SOHO 2026) - "Avapritinib was deferred due to hemorrhagic risk. SM-aCML is a high-risk, clonally unified myeloid neoplasm with marked proliferative potential. This case defines three actionable principles: (1) sTLS may occur at high leukocyte burdens, warranting early metabolic monitoring; (2) ASXL1/TET2 mutations identify aggressive disease biology; (3) severe thrombocytopenia favors azacitidine-first cytoreduction prior to KIT inhibition. These findings provide a practical molecular and clinical framework for this rare entity."

Clinical • Acute Myelogenous Leukemia • Atypical Chronic Myeloid Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Neuroblastoma • Oncology • Solid Tumor • ABL1 • ASXL1 • BCR • IL2RA • NRAS • TET2

Circulating tumor DNA (ctDNA) analyses of the phase III VOYAGER trial: KIT mutational landscape and outcomes in patients with advanced gastrointestinal stromal tumor (GIST). (ASCO 2022) - P3 | "Background: The genotype of primary mutations predicts imatinib response in untreated metastatic GIST...Regorafenib showed similar activity regardless of KIT mutational status and the location of KIT mutation... Hybrid capture-based plasma sequencing detects ctDNA in the majority of patients with advanced TKI-resistant GIST, including heterogeneity of KIT mutations. This study is the first to show that ctDNA sequencing correlates with outcomes in pretreated GIST. Identification of ABP (exon13/14) KIT mutations negatively correlates with avapritinib activity."

Circulating tumor DNA • Clinical • P3 data • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

Circulating tumor DNA analysis of the phase III VOYAGER trial: KIT mutational landscape and outcomes in patients with advanced gastrointestinal stromal tumor treated with avapritinib or regorafenib. (PubMed, Ann Oncol) - P3 | "CtDNA sequencing efficiently detects KIT/PDGFRA mutations and prognosticates outcomes in patients with TKI-resistant GIST treated with avapritinib. ctDNA analysis can be used to monitor disease progression and provide more personalized treatment."

Circulating tumor DNA • Journal • Metastases • P3 data • Stroma • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

MRD-positive AML Clinical Study (clinicaltrials.gov) - P=N/A | N=537 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | N=120 ➔ 537 | Trial completion date: Apr 2028 ➔ Oct 2028 | Trial primary completion date: May 2026 ➔ May 2028

Enrollment change • Minimal residual disease • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • DEK • FLT3 • NPM1 • NUP214 • RUNX1 • RUNX1T1

Precision therapeutic strategies for advanced gastrointestinal stromal tumors. (PubMed, Cancer Metastasis Rev) - "Imatinib, followed by later-line tyrosine kinase inhibitors, including sunitinib, regorafenib, ripretinib, and genotype-selected avapritinib, have substantially prolonged survival. In this review, we summarize the biological and molecular landscape of advanced GIST, as well as the current therapeutic strategies and major mechanisms of resistance. Finally, we highlight promising therapeutic strategies supported by preclinical and clinical evidence that may advance subtype-informed precision medicine."

Journal • Review • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

Avapritinib leads to sustained improvement in skin findings in patients with indolent systemic mastocytosis (ISM) with a well-tolerated safety profile: Long-term analyses from the PIONEER study (ADO 2026) - No abstract available

Clinical • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Solid Tumor

Avapritinib leads to sustained improvement in skin findings in patients with indolent systemic mastocytosis (ISM) with a well-tolerated safety profile: Long-term analyses from the PIONEER study (ADO 2026) - No abstract available

Clinical • Oncology

Avapritinib improves symptoms and quality of life in indolent systemic mastocytosis: 12-month outcome of the real-world AVATAR study (EADV 2026) - No abstract available

Clinical • HEOR • Real-world • Real-world evidence • Cardiovascular • Dermatology • Dermatopathology • Immunology • Urticaria

Symptom Severity Reduction and QoL Improvement With Avapritinib in Indolent Systemic Mastocytosis Through 4 Years (ACAAI 2026) - No abstract available

Late-breaking abstract

Efficacy and safety analysis of neoadjuvant avapritinib in patients with platelet-derived growth factor receptor α D842V-mutant gastrointestinal stromal tumors (PubMed, Zhonghua Wei Chang Wai Ke Za Zhi) - " Avapritinib as neoadjuvant therapy in patients with PDGFRA-D842V-mutant gastrointestinal stromal tumors demonstrates significant efficacy in reducing tumor size and improving the rate of complete resection. Despite a relatively high incidence of adverse events, the treatment exhibits a manageable safety profile and is generally well tolerated."

Journal • Gastric Cancer • Gastrointestinal Stromal Tumor • Hematological Disorders • Leukopenia • Oncology • Sarcoma • Solid Tumor • PDGFRA

Combinatorial Targeting of Avapritinib-Driven MAP Kinase Activation in High-Grade Glioma (SNO 2026) - No abstract available

Brain Cancer • Glioma • High Grade Glioma • Solid Tumor

Avapritinib As First-Line Therapy in Patients with Advanced Systemic Mastocytosis: Efficacy and Safety from the Pathfinder Clinical Study (ASH 2022) - P1, P2 | "Avapritinib demonstrated a high level of efficacy as the first-line therapy for patients with AdvSM across all disease subtypes with an ORR of 84% and an OS rate of 88% at 2 years. Avapritinib treatment with a 200 mg once-daily starting dose was generally well-tolerated."

Clinical • Anemia • Hematological Disorders • Hematological Malignancies • Immunology • Myeloproliferative Neoplasm • Neutropenia • Oncology • Thrombocytopenia

Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. (PubMed, Nat Med) - P1 | "Avapritinib elicited ≥50% reductions in marrow mast cells and serum tryptase in 92% and 99% of patients, respectively. Avapritinib induced deep and durable responses, including molecular remission of KIT D816V in patients with AdvSM, and was well tolerated at the recommended phase 2 dose of 200 mg daily."

Clinical • Journal • P1 data • Hematological Disorders • Hematological Malignancies • Immunology • Oncology • Thrombocytopenia

AVAPRITINIB IN PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM): EFFICACY AND SAFETY ANALYSIS FROM THE PHASE 2 PATHFINDER STUDY WITH 3-YEAR FOLLOW-UP (EHA 2024) - P1, P2 | "With >3 years of follow-up, patients with AdvSM treated with avapritinib showed continued deep and durableresponses with a favorable benefit-risk profile regardless of AdvSM subtype or prior therapy."

Clinical • Metastases • P2 data • Aggressive Systemic Mastocytosis • Anemia • Eosinophilia • Hematological Disorders • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia

Prospective Study of Avapritinib in Patients with Relapsed/Refractory or MRD-Positive Core-Binding Factor Acute Myeloid Leukemia with KIT Mutations (ASH 2024) - "In general, the study results suggest that avapritinib demonstrated good efficacy and safety in patients with CBF-AML harboring KIT gene mutations. These findings may support expanding the indications for this therapy in the treatment of AML."

Clinical • Minimal residual disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Gastrointestinal Cancer • Gastrointestinal Stromal Tumor • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Respiratory Diseases • Sarcoma • CBFB • KIT • PDGFRA • RUNX1 • RUNX1T1

THE REVISED MUTATION-ADJUSTED RISK SCORE (MARS-R) FOR PREDICTING OVERALL SURVIVAL IN PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS TREATED WITH MIDOSTAURIN OR AVAPRITINIB (EHA 2025) - P1, P2 | "The MARS-R captures the continuum of OS risk observed among AdvSM pts, stratifying them into 3 distinct risk categories for treatment with midostaurin or avapritinib. It serves as an important new tool for clinical management of AdvSM patients, particularly regarding treatment maintenance or intensification, such as with allogeneic hematopoietic cell transplantation."

Clinical • Metastases • Aggressive Systemic Mastocytosis • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Oncology • ASXL1 • RUNX1 • SETBP1

Efficacy and safety of avapritinib in advanced systemic mastocytosis: 4-year follow-up of the PATHFINDER study. (PubMed, Blood Adv) - P2 | "Eleven (10%) patients experienced TEAEs leading to death, of which 1 was deemed related to avapritinib by the principal investigator. With 4-year follow-up, avapritinib-treated patients with AdvSM experienced deep and durable responses and a favorable benefit-risk profile."

Journal • Hematological Disorders • Hematological Malignancies • Oncology • Thrombocytopenia • KIT

EFFICACY AND SAFETY OF BEZUCLASTINIB IN PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS: PRIMARY RESULTS FROM THE APEX STUDY (EHA 2026) - "In the ITT population, median age (range) was 70 (43-87) years; 23 (28%) patients were female; 28 patients (35%; avapritinib, 7%; midostaurin, 30%) received prior KIT-targeted TKI therapy. Summary/Conclusion Bezuclastinib demonstrated high response rates, led to deep reductions in objective markers of MC burden, and was well-tolerated, with infrequent dose reductions and no discontinuations due to TRAEs. Positive results from Apex support bezuclastinib as a potential treatment for patients with AdvSM."

Clinical • Metastases • Aggressive Systemic Mastocytosis • Hematological Disorders • Hematological Malignancies • Leukemia • Mast Cell Leukemia • Myeloproliferative Neoplasm • Neutropenia • Thrombocytopenia

Reversal of systemic mastocytosis-induced liver cirrhosis with avapritinib treatment: case report. (PubMed, Leuk Lymphoma) - No abstract available

Journal • Fibrosis • Gastroenterology • Hepatology • Immunology • Liver Cirrhosis

Waxing and Waning Mediastinal Lymphadenopathy and Osteosclerosis in Systemic Mastocytosis: Diagnostic Vigilance During Targeted Therapy (SOHO 2026) - "Subsequently, PET-CT demonstrated complete nodal quiescence.With a peak tryptase of 242 μg/L and KIT D816V allele burden of 90%, avapritinib 25 mg daily was initiated, achieving marked tryptase reduction to 67.6 μg/L with concurrent bone scan improvement, confirming meaningful biologic response in both mast cell burden and osteosclerotic disease activity... This case demonstrates the need for multidisciplinary vigilance in SM patients on targeted therapy and highlights that osteosclerotic improvement and tryptase reduction alone do not preclude concurrent aggressive nodal progression or transformation. CT: computed tomography, CTA: computed tomography angiography, PET-CT: positron emission tomography–computed tomography, SUV: standardized uptake value."

Hematological Malignancies • Lymphoma • Oncology

KIT D816V-Selective Tyrosine Kinase Inhibitors for the Treatment of Advanced Systemic Mastocytosis: A Systematic Review and Meta-Analysis of Efficacy and Safety Outcomes (SOHO 2026) - "Our findings reveal that avapritinib delivers the highest ORRs and deepest clinical remissions for AdvSM, reinforcing its role as a preferred frontline therapy. However, its significant association with severe neutropenia demands strict baseline patient selection and continuous hematologic monitoring. Alternatively, midostaurin and the investigational TKI bezuclastinib offer favorable safety profiles and may serve as preferable options for patients with a high baseline risk of hematologic toxicities."

Metastases • Retrospective data • Review • Oncology