Trial reportJournal of cancer research and clinical oncology2015
Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study.
Trial report in Journal of cancer research and clinical oncology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00109707 (A Phase IA/II Multicenter, Dose-escalation Study of Oral AMN107 on a Continuous Daily Dosing Schedule in Adult Patients With Imatinib-resistant/Intolerant CML in Chronic or Accelerated Phase or Blast Crisis, Relapsed/Refractory Ph+ ALL, and Other Hematologic Malignancies.), which is not on this map. Cited by 30 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase IA/II Multicenter, Dose-escalation Study of Oral AMN107 on a Continuous Daily Dosing Schedule in Adult Patients With Imatinib-resistant/Intolerant CML in Chronic or Accelerated Phase or Blast Crisis, Relapsed/Refractory Ph+ ALL, and Other Hematologic Malignancies.
Who cites it
30 citing papers in PubMed, 53 citations in OpenAlex.
- Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial.Leukemia · 2018Trial
- Unraveling the Rare Entity ofJournal of hematology · 2024Article
- Acute mast cell leukemia without KIT D816V mutation and lack of CD2 and CD25-a case report of rare entity.Journal of hematopathology · 2023Article
- Avapritinib for the treatment of KIT mutation-negative systemic mastocytosis.Proceedings (Baylor University. Medical Center) · 2023Article
- c-Kit Receptors as a Therapeutic Target in Cancer: Current Insights.OncoTargets and therapy · 2023Review
- Understanding human mast cells: lesson from therapies for allergic and non-allergic diseases.Nature reviews. Immunology · 2022Review
- Treatment of Indolent and Advanced Systemic Mastocytosis.Mediterranean journal of hematology and infectious diseases · 2022Review
- Precision Medicine in Systemic Mastocytosis.Medicina (Kaunas, Lithuania) · 2021Review
- Mastocytosis and Mast Cell Activation Disorders: Clearing the Air.International journal of molecular sciences · 2021Review
- Avapritinib in the Treatment of Systemic Mastocytosis: an Update.Current hematologic malignancy reports · 2021Review
- Review
- Avapritinib for Systemic Mastocytosis.Expert review of hematology · 2021Article
- New Insights into the Pathogenesis of Systemic Mastocytosis.International journal of molecular sciences · 2021Review
- Response Criteria in Advanced Systemic Mastocytosis: Evolution in the Era of KIT Inhibitors.International journal of molecular sciences · 2021Review
- Systemic Mastocytosis: Molecular Landscape and Implications for Treatment.Mediterranean journal of hematology and infectious diseases · 2021Review
- Comparative aspects of mast cell neoplasia in animals and the role of KIT in prognosis and treatment.Veterinary medicine and science · 2020Review
- Systemic Mastocytosis: Following the Tyrosine Kinase Inhibition Roadmap.Frontiers in pharmacology · 2020Review
- Comparative oncology: The paradigmatic example of canine and human mast cell neoplasms.Veterinary and comparative oncology · 2019Review
- Novel approaches to treating advanced systemic mastocytosis.Clinical pharmacology : advances and applications · 2019Review
- The new tool "Hematology. American Society of Hematology. Education Program · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 8 institutions in 4 countries.
Funding
Abstract
purposeActivating KIT mutations are part of the pathogenesis of systemic mastocytosis (SM). Nilotinib is a tyrosine kinase inhibitor that potently inhibits activated forms of KIT. This phase 2, open-label, single-arm study (CAMN107A2101; www.clinicaltrials.gov NCT00109707) evaluated nilotinib in patients with SM.
methodsPatients with SM [aggressive SM (ASM), indolent SM, or other] received nilotinib 400 mg twice daily. C-findings were collected retrospectively to assess response using criteria proposed after trial initiation. Response was evaluated using improvements in laboratory findings (for all patients) and ASM response criteria (for the ASM subgroup).
resultsIn 61 patients enrolled, the median nilotinib exposure was 232 days (range 3-1274 days) with a median follow-up of 34.7 months. In patients with ASM (n = 37), the overall response rate was 21.6 %. In the eight responders, all of whom had a KIT D816V mutation at any time, mast cell infiltration and tryptase level decreased by 70 % and 29.8 %, respectively; absolute neutrophil count increased by 94.7 %. Laboratory parameters also improved in the non-ASM subgroups. Overall survival at 24 months was 81.2 % (95 % CI 70.6-91.8 %) with median survival not yet reached. New or worsening grade 3/4 hematologic adverse events (AEs) included thrombocytopenia (10.3 %), anemia (10.0 %), and neutropenia (6.9 %). The most common grade 3/4 nonhematologic drug-related AEs were diarrhea (6.6 %) and headache (4.9 %). Eleven patients (9 with ASM, 2 with MCL) died, 10 due to progressive disease; 7 deaths occurred ≥28 days after treatment discontinuation.
conclusionsNilotinib 400 mg twice daily was effective in some patients with SM, including patients with mutated KIT D816V.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.