Abstract
Spontaneous pneumothorax is a common respiratory presentation that may signal underlying genetic disease. Familial pneumothorax occurs in ~10% of primary cases, yet 75% remain genetically unclassified. We report identical twin brothers presenting with spontaneous pneumothoraces in adulthood, leading to a diagnosis of Tatton-Brown-Rahman syndrome (TBRS), a DNMT3A-related overgrowth disorder not previously associated with pneumothorax. Both individuals exhibited tall stature, mild intellectual disability, hypermobility, and cardiac abnormalities. Whole genome sequencing identified a rare de novo DNMT3A missense variant (c.1585 G > A, p.D529N) absent from population databases and predicted to be damaging. Methylation profiling confirmed genome-wide hypomethylation consistent with impaired DNMT3A function, supporting pathogenicity. No variants were found in known familial pneumothorax genes. Apical blebs observed at surgery and connective tissue features suggest a mechanistic link between TBRS and pneumothorax, analogous to other monogenic connective tissue disorders. This case expands the phenotypic spectrum of TBRS and highlights the importance of genetic evaluation in familial pneumothorax. Diagnosis enables personalised care, including surveillance for extrapulmonary complications such as aortic root dilatation and haematological malignancy. Our findings suggest that TBRS should be considered in patients presenting with pneumothorax, tall stature, and neurodevelopmental features. Further cases are needed to confirm this association and refine clinical management strategies.
| Original language | English |
|---|---|
| Pages (from-to) | 723-726 |
| Number of pages | 4 |
| Journal | European Journal of Human Genetics |
| Volume | 34 |
| Issue number | 5 |
| Early online date | 25 Feb 2026 |
| DOIs | |
| Publication status | Published - 1 May 2026 |
Bibliographical note
Copyright © The Author(s) 2026. This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit https://creativecommons.org/licenses/by/4.0/.Data Access Statement
Data that support the findings of this study are available on request from the corresponding author with reasonable patient privacy restrictions.Funding
We thank the Medical Research Council (SJM), NIHR Cambridge Biomedical Research Centre (BRC-1215-20014) (SJM, ERM), LifeArc (SJM), EpiGenRare MRC Rare Diseases Node funding MR/Y008170/1 (ERM), Rosetrees Trust (SL, ERM), Royal Papworth Hospital (SJM), and the Alpha1-Foundation (SJM).
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