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Patients with monogenic immune dysregulation show improvement in childhood interstitial lung disease following targeted therapy -- a case series and single center approach
  • +11
  • Holly Wobma,
  • Ryan Perkins,
  • Lisa Bartnikas,
  • Fatma Dedeoglu,
  • Janet Chou,
  • Ruth Ann Vleugels,
  • Mindy Lo,
  • Erin Janssen,
  • Lauren Henderson,
  • Jennifer Whangbo,
  • Sara Vargas,
  • Martha Fishman,
  • Katie Krone,
  • Alicia Casey
Holly Wobma
Boston Children's Hospital

Corresponding Author:[email protected]

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Ryan Perkins
Boston Children's Hospital Division of Pulmonary and Respiratory Diseases
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Lisa Bartnikas
Boston Children's Hospital Division of Immunology
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Fatma Dedeoglu
Boston Children's Hospital Division of Immunology
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Janet Chou
Boston Children's Hospital Division of Immunology
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Ruth Ann Vleugels
Brigham and Women's Hospital Department of Dermatology
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Mindy Lo
Boston Children's Hospital Division of Immunology
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Erin Janssen
Boston Children's Hospital Division of Immunology
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Lauren Henderson
Boston Children's Hospital Division of Immunology
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Jennifer Whangbo
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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Sara Vargas
Boston Children's Hospital Department of Pathology
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Martha Fishman
Boston Children's Hospital Division of Pulmonary and Respiratory Diseases
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Katie Krone
Boston Children's Hospital Division of Pulmonary and Respiratory Diseases
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Alicia Casey
Boston Children's Hospital Division of Pulmonary and Respiratory Diseases
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Abstract

In recent years, a number of monogenic disorders have been described that are characterized by immune dysregulation. A subset of these ‘primary immune regulatory disorders’ can cause severe interstitial lung disease, often recognized in late childhood or adolescence. Patients presenting to pulmonary clinic may have long and complex medical histories but lack a unifying genetic diagnosis. It is crucial for pulmonologists to recognize features suggestive of multisystem immune dysregulation and to initiate genetic workup, since targeted therapies based on underlying genetics may halt or even reverese pulmonary disease progression. Through such an approach, our center has been able to diagnose and treat a cohort of patients with interstitial lung disease from gene defects that affect immune regulation. Here we present representative cases related to pathogenic mutations in three distinct pathways and summarize disease manifestations and treatment approaches. We conclude with a discussion of our perspective on the outstanding challenges for diagnosing and managing these complex life-threatening and chronic disorders.
11 Dec 2021Submitted to Pediatric Pulmonology
11 Dec 2021Submission Checks Completed
11 Dec 2021Assigned to Editor
12 Dec 2021Reviewer(s) Assigned
19 Jan 2022Review(s) Completed, Editorial Evaluation Pending
19 Jan 2022Editorial Decision: Revise Major
23 Mar 20221st Revision Received
24 Mar 2022Submission Checks Completed
24 Mar 2022Assigned to Editor
24 Mar 2022Reviewer(s) Assigned
04 Apr 2022Review(s) Completed, Editorial Evaluation Pending
04 Apr 2022Editorial Decision: Accept