Evidence map›Paper›PMID 27527645›Full record

GuidelineNeurology2016

Sleep and pulmonary outcomes for clinical trials of airway plexiform neurofibromas in NF1.

Scott R Plotkin, Stephanie D Davis, Kent A Robertson, Srivandana Akshintala, Julian Allen, Michael J Fisher, Jaishri O Blakeley, Brigitte C Widemann, Rosalie E Ferner, Carole L Marcus and 1 more

Open access · bronzeAbstract readConsensus StatementPractice Guideline
PubMed Full text at PMC Publisher
In one paragraph

Guideline in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
0.4field-weighted citation impact, top 32% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

13 citing papers in PubMed, 19 citations in OpenAlex.

  1. Trial
  2. Selumetinib in Children with Inoperable Plexiform Neurofibromas.The New England journal of medicine · 2020
    Trial
  3. Article
  4. Review
  5. Review
  6. Article
  7. Article
  8. Review
  9. Review
  10. Article
  11. Article
  12. AfterNeuro-oncology advances · 2020
    Review
  13. Review
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

11 authors at 8 institutions in 2 countries.

Scott R PlotkinFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK. splotkin@partners.org.
Stephanie D DavisFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Kent A RobertsonFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Srivandana AkshintalaFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Julian AllenFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Michael J FisherFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Jaishri O BlakeleyFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Brigitte C WidemannFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Rosalie E FernerFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
Carole L MarcusFrom the Neurology Department and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Section of Pediatric Pulmonology, Allergy and Sleep Medicine (S.D.D.), and Stem Cell Transplantation Program (K.A.R.), Riley Children's Hospital, Indiana University School of Medicine, Indianapolis; Pediatric Oncology Branch (S.A., B.C.W.), National Cancer Institute, Bethesda, MD; Division of Pulmonary Medicine (J.A.), Division of Oncology (M.J.F.), and Sleep Center (C.L.M.), Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.) and Sleep Center (C.L.M.), The Perelman School of Medicine at the University of Pennsylvania (J.A.), Philadelphia; Department of Neurology (J.O.B.), John Hopkins Medical Institute, Baltimore, MD; and Department of Neurology (R.E.F.), Guy's and St. Thomas' NHS Foundation Trust and Institute of Psychiatry, King's College London, UK.
REiNS International Collaboration
Massachusetts General Hospital · USKing's College London · GBUniversity of Pennsylvania · USGuy's and St Thomas' NHS Foundation Trust · GBChildren's Hospital of Philadelphia · USNational Cancer Institute · USRiley Hospital for Children · USIndiana University – Purdue University Indianapolis · US

Funding

Therapy for NF1-Related Tumors and other Genetic Tumor Predisposition SyndromesZIABC010801 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2020
$13.8M
Mouse Neurodevelopmental Behavior CoreU54HD090255 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI SAHIN, MUSTAFA · 2016 to 2020
$5.2M
NICHD NIH HHS U54 HD090255
6 · The paper itself

Abstract

objectivePlexiform neurofibromas (PNs) are complex, benign nerve sheath tumors that occur in approximately 25%-50% of individuals with neurofibromatosis type 1 (NF1). PNs that cause airway compromise or pulmonary dysfunction are uncommon but clinically important. Because improvement in sleep quality or airway function represents direct clinical benefit, measures of sleep and pulmonary function may be more meaningful than tumor size as endpoints in therapeutic clinical trials targeting airway PN.

methodsThe Response Evaluation in Neurofibromatosis and Schwannomatosis functional outcomes group reviewed currently available endpoints for sleep and pulmonary outcomes and developed consensus recommendations for response evaluation in NF clinical trials.

resultsFor patients with airway PNs, polysomnography, impulse oscillometry, and spirometry should be performed to identify abnormal function that will be targeted by the agent under clinical investigation. The functional group endorsed the use of the apnea hypopnea index (AHI) as the primary sleep endpoint, and pulmonary resistance at 10 Hz (R10) or forced expiratory volume in 1 or 0.75 seconds (FEV1 or FEV0.75) as primary pulmonary endpoints. The group defined minimum changes in AHI, R10, and FEV1 or FEV0.75 for response criteria. Secondary sleep outcomes include desaturation and hypercapnia during sleep and arousal index. Secondary pulmonary outcomes include pulmonary resistance and reactance measurements at 5, 10, and 20 Hz; forced vital capacity; peak expiratory flow; and forced expiratory flows.

conclusionsThese recommended sleep and pulmonary evaluations are intended to provide researchers with a standardized set of clinically meaningful endpoints for response evaluation in trials of NF1-related airway PNs.

Indexed as

Clinical Trials as TopicHumansLungNeurofibroma, PlexiformNeurofibromatosis 1OscillometryPolysomnographySleepSpirometryTreatment Outcome

Identifiers

PMID27527645
PMCPMC5578358
OpenAlexW2461954968

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.