Evidence map›Paper›PMID 28744009›Full record

Trial reportLeukemia2018

Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial.

D J DeAngelo, T I George, A Linder, C Langford, C Perkins, J Ma, P Westervelt, J D Merker, C Berube, S Coutre and 8 more

Registry-linked trialAbstract readClinical Trial, Phase II
PubMed Publisher
In one paragraph

Trial report in Leukemia, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00233454 (A Single Arm, Phase 2, Open-Label Study to Determine the Efficacy of Twice Daily Oral Dosing of PKC412 <Midostaurin> Administered to Patients With Aggressive Systemic Mastocytosis), which is not on this map. Cited by 68 papers.

0numbers the graph read from it
0cells of the map it votes in
68citing papers in PubMed
5.3field-weighted citation impact, top 4% of its field
1 · What the graph read from it

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.

2 · The registry

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.

NCT00233454 phase2completednot on this map

A Single Arm, Phase 2, Open-Label Study to Determine the Efficacy of Twice Daily Oral Dosing of PKC412 <Midostaurin> Administered to Patients With Aggressive Systemic Mastocytosis (ASM) and Mast Cell Leukemia (MCL)

TypeinterventionalSponsorJason Robert GotlibRan2005 to 2011Enrolled26ConditionsSystemic Mastocytosis, Aggressive (ASM), Leukemia, Mast Cell, Hematological Non-mast Cell Lineage Disease (AHNMD)ArmsMidostaurin
3 · Its place in the literature

Who cites it

68 citing papers in PubMed, 138 citations in OpenAlex.

  1. Article
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  3. Review
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  5. Article
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  11. Article
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  14. Article
  15. Review
  16. Review
  17. Review
  18. Systemic Mastocytosis: State of the Art.Current hematologic malignancy reports · 2024
    Review
  19. Article
  20. Review

8 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors at 9 institutions in 2 countries.

D J DeAngeloDepartment of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.
T I GeorgeDepartment of Pathology, University of New Mexico, Albuquerque, NM, USA.
A LinderDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
C LangfordDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
C PerkinsDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
J MaDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
P WesterveltDivision of Hematology/Oncology, Washington University School of Medicine, St Louis, MO, USA.
J D MerkerDepartment of Pathology, Stanford University School of Medicine, Stanford, CA, USA.
C BerubeDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
S CoutreDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
M LiedtkeDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
B MedeirosDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
D SternbergNovartis Pharmaceuticals, Florham Park, NJ, USA.
C DutreixNovartis Pharmaceuticals, Florham Park, NJ, USA.
P-A RuffieNovartis Pharmaceuticals, Florham Park, NJ, USA.
C CorlessDepartment of Pathology, Oregon Health and Sciences University, Portland, OR, USA.
T J GraubertDivision of Hematology/Oncology, Department of Medicine, Massachusetts General Hospital, Boston, MA, USA.
J GotlibDivision of Hematology, Department of Medicine, Stanford University School of Medicine/Stanford Cancer Institute, Stanford, CA, USA.
Stanford Cancer InstituteStanford University · USNovartis (Switzerland) · CHDana-Farber Cancer Institute · USMassachusetts General Hospital · USNovartis (United States) · USOregon Health & Science University · USUniversity of New Mexico · USWashington University in St. Louis · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Patients with advanced systemic mastocytosis (SM) (e.g. aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL)) have limited treatment options and exhibit reduced survival. Midostaurin is an oral multikinase inhibitor that inhibits D816V-mutated KIT, a primary driver of SM pathogenesis. We conducted a phase II trial of midostaurin 100 mg twice daily, administered as 28-day cycles, in 26 patients (ASM, n=3; SM-AHN, n= 17; MCL, n=6) with at least one sign of organ damage. During the first 12 cycles, the overall response rate was 69% (major/partial response: 50/19%) with clinical benefit in all advanced SM variants. With ongoing therapy, 2 patients achieved a complete remission of their SM. Midostaurin produced a ⩾50% reduction in bone marrow mast cell burden and serum tryptase level in 68% and 46% of patients, respectively. Median overall survival for the entire cohort was 40 months, and 18.5 months for MCL patients. Low-grade gastrointestinal side effects were common and manageable with antiemetics. The most frequent grade 3/4 nonhematologic and hematologic toxicities were asymptomatic hyperlipasemia (15%) and anemia (12%). With median follow-up of 10 years, no unexpected toxicities emerged. These data establish the durable activity and tolerability of midostaurin in advanced SM.

Indexed as

AdultAgedFemaleFollow-Up StudiesHumansLeukemia, Mast-CellMaleMastocytosis, SystemicMiddle AgedProtein Kinase InhibitorsStaurosporineYoung AdultmidostaurinProtein Kinase InhibitorsStaurosporine

Identifiers

PMID28744009
OpenAlexW2736420338

What OpenQuestion holds

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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.