Evidence map›Paper›PMID 36459725›Full record

ArticleInternational journal of pediatric otorhinolaryngology2023

Primary targeted medical therapy for management of bilateral head and neck lymphatic malformations in infants.

Clare M Richardson, Jonathan N Perkins, Kaitlyn Zenner, Catherine Bull, Erika Lutsky, Dana M Jensen, Victoria Dmyterko, James T Bennett, Tara L Wenger, John P Dahl and 4 more

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of pediatric otorhinolaryngology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.1field-weighted citation impact, top 22% 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

3 citing papers in PubMed, 5 citations in OpenAlex.

  1. Multidimensional outcomes of initial treatment strategies for pediatric head and neck lymphatic malformations.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026
    Article
  2. Treatments and Outcomes of Pediatric Head and Neck Lymphatic Malformations: A 20-Year Single Institution Experience.Otolaryngology--head and neck surgery : official journal of American Academy of Otolaryngology-Head and Neck Surgery · 2025
    Article
  3. 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

14 authors at 3 institutions in 1 country.

Clare M RichardsonDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA. Electronic address: clare.richardson@seattlechildrens.org.
Jonathan N PerkinsDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA. Electronic address: jonathan.perkins2@seattlechildrens.org.
Kaitlyn ZennerDepartment of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA. Electronic address: kzenner@uw.edu.
Catherine BullDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA. Electronic address: cathy.bull@seattlechildrens.org.
Erika LutskyDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA. Electronic address: erika.lutsky@seattlechildrens.org.
Dana M JensenCenter for Clinical and Translational Research, Seattle Children's Hospita, USAl. Electronic address: dana.jensen@seattlechildrens.org.
Victoria DmyterkoCenter for Clinical and Translational Research, Seattle Children's Hospita, USAl. Electronic address: Victoria.dmyterko@seattlechildrens.org.
James T BennettCenter for Clinical and Translational Research, Seattle Children's Hospita, USAl; Division of Genetic Medicine, Department of Pediatrics, Seattle Children's Hospital, USA. Electronic address: james.bennett@seattlechildrens.org.
Tara L WengerDivision of Genetic Medicine, Department of Pediatrics, Seattle Children's Hospital, USA. Electronic address: tara.wenger@seattlechildrens.org.
John P DahlDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA; Center for Clinical and Translational Research, Seattle Children's Hospita, USAl. Electronic address: jake.dahl@seattlechildrens.org.
Juliana Bonilla-VelezDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA; Center for Clinical and Translational Research, Seattle Children's Hospita, USAl. Electronic address: juliana.bonilla-velez@seattlechildrens.org.
Randall A BlyDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA. Electronic address: randall.bly@seattlechildrens.org.
Amy E GeddisCenter for Clinical and Translational Research, Seattle Children's Hospita, USAl; Division of Hematology-Oncology, Department of Pediatrics, Seattle Children's Hospital, USA. Electronic address: amy.geddis@seattlechildrens.org.
Jonathan A PerkinsDivision of Otolaryngology - Head and Neck Surgery, Department of Surgery, Seattle Children's Hospital, MS OA.9.220, PO Box 5371, Seattle, WA, 98145, USA; Department of Otolaryngology - Head and Neck Surgery, University of Washington, 1959 NE Pacific St, Box 356515, Seattle, WA, 98195, USA; Center for Clinical and Translational Research, Seattle Children's Hospita, USAl. Electronic address: Jonathan.Perkins@seattlechildrens.org.
Seattle Children's Hospital · USSeattle Children's Hospital · USUniversity of Washington · US

Funding

Research Training in OtolaryngologyT32DC000018 · NIDCD · UNIVERSITY OF WASHINGTON · PI STONE, JENNIFER S. · 1989 to 2023
$7.8M
Mosaic: post-zygotic mutations in vascular malformationsR01HL130996 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI BENNETT, JAMES T · 2016 to 2024
$7.5M
Characterization of the PIK3CA mutation spectrum in lymphatic malformationsF32HL147398 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI ZENNER, KAITLYN · 2019 to 2019
$64k
NHLBI NIH HHS F32 HL147398NHLBI NIH HHS R01 HL130996NIDCD NIH HHS T32 DC000018
6 · The paper itself

Abstract

objectivesPatients born with bilateral head and neck lymphatic malformations (BHNLMs) often require multiple invasive treatments, including tracheostomy. We hypothesized that primary targeted medical therapy (pTMT) with diagnostic needle aspiration reduces the need for invasive therapy such as surgical resection and/or sclerotherapy.

methodsRetrospective case review was performed of infants with BHNLMs (Grade 2 or De Serres stage IV and V) treated only at our institution from 2000 to 2021. Patients were divided into two cohorts: those managed with pTMT and those managed with observation, sclerotherapy, or surgical intervention (non-pTMT). Data regarding interventions, clinical outcomes, morbidity, and mortality were analyzed with descriptive statistics.

resultsNine children with BHNLMs met inclusion criteria. Three (33%) were in the pTMT cohort and six (66%) were non-pTMT. Eight (89%) malformations were genotyped, and all demonstrated hotspot PIK3CA variants. All pTMT patients had sirolimus initiated in the first month of life and underwent needle aspiration of malformation cyst fluid for cell-free DNA samples. All pTMT patients tolerated medical therapy. For the non-pTMT cohort, primary treatment included none (deceased, n = 1, 17%), observation with needle aspiration (n = 1, 17%), surgical resection (n = 2, 33%), or combination surgery and sclerotherapy (n = 2, 33%). Intubation duration, intensive care and initial hospital length of stay were not different between cohorts. Four non-pTMT patients (67%) required tracheostomy, and two (33%) died prior to discharge. All pTMT patients survived and none required tracheostomy. Non-pTMT patients required a median of two invasive therapies prior to discharge (IQR 1-4) and a mean total of 13 over the course of their lifetime (IQR 1-16), compared to the pTMT group who did not require any lifetime invasive therapy, even after initial pTMT and discharge home.

conclusionThis study compares patients with BHNLMs (Grade 2) treated with pTMT versus those treated with observation or invasive therapy. Patients treated with pTMT required no surgical or invasive procedural treatment of their malformations, no tracheostomy placement, no unplanned readmissions after discharge, and had no mortalities. Needle aspiration was useful as a therapeutic adjunct for cell-free DNA diagnosis of PIK3CA variants, which guided TMT.

Indexed as

HeadLymphatic AbnormalitiesChildHumansInfantNeckRetrospective StudiesSclerotherapyTreatment OutcomeLymphatic malformationNeedle aspirationPI3KSurgeryTargeted therapyTreatment

Identifiers

PMID36459725
PMCPMC10243723
OpenAlexW4308987275

What Socratic holds

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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 Socratic graph.