Evidence map›Paper›PMID 42012601›Full record

ReviewMolecular and cellular pediatrics2026

Diagnoses of double heterozygous NF1 variants and dual RASopathy.

Daniela Angelova-Toshkina, Denny Schanze, Pia Vaassen, Michael C Frühwald, Thorsten Rosenbaum, Martin Zenker, Michaela Kuhlen

Abstract readReview
In one paragraph

Review in Molecular and cellular pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

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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

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3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

7 authors.

Daniela Angelova-ToshkinaPediatrics and Adolescent Medicine, Faculty of Medicine, University of Augsburg, Stenglinstr. 2, Augsburg, 86156, Germany.
Denny SchanzeInstitute of Human Genetics, Otto-von-Guericke-University Magdeburg, Leipziger Str. 44, Magdeburg, 39120, Germany.
Pia VaassenDepartment of Pediatrics, Sana Kliniken Duisburg, Zu den Rehwiesen 9, Duisburg, 47055, Germany.
Michael C FrühwaldPediatrics and Adolescent Medicine, Faculty of Medicine, University of Augsburg, Stenglinstr. 2, Augsburg, 86156, Germany.
Thorsten RosenbaumDepartment of Pediatrics, Sana Kliniken Duisburg, Zu den Rehwiesen 9, Duisburg, 47055, Germany.
Martin Zenker *Institute of Human Genetics, Otto-von-Guericke-University Magdeburg, Leipziger Str. 44, Magdeburg, 39120, Germany.
Michaela Kuhlen *Pediatrics and Adolescent Medicine, Faculty of Medicine, University of Augsburg, Stenglinstr. 2, Augsburg, 86156, Germany. michaela.kuhlen@uk-augsburg.de.

Funding

Deutsche Forschungsgemeinschaft KU 3764/3-1Deutsche Krebshilfe 70115888
6 · The paper itself

Abstract

Genetic testing in neurofibromatosis type 1 (NF1) occasionally reveals two heterozygous NF1 variants in the same individual. Correct interpretation hinges on allelic phasing, elucidation of somatic second hits in lesions, and distinction from bona fide dual RASopathy diagnoses. We provide a focused, critical review of the primary literature on (i) cis doublets in NF1, (ii) purported germline trans configurations—particularly in spinal neurofibromatosis, (iii) biallelic somatic inactivation of NF1 in café-au-lait macules, tumors, and juvenile myelomonocytic leukemia, and (iv) confirmed cases of dual RASopathy. We also include an index patient with two heterozygous NF1 variants to illustrate clinical challenges. Phase-proven cis doublets are rare, often closely spaced, and generally accompany classic NF1 without reproducible phenotype escalation. Claims of germline trans NF1 pathogenic variants weaken on reappraisal: second alleles reported in spinal neurofibromatosis typically do not meet current pathogenicity standards, while Nf1−/− mouse embryonic lethality underscores the biological implausibility of constitutive biallelic loss. In contrast, biallelic somatic NF1 inactivation is pervasive across NF1 lesions, including café-au-lait macules, plexiform and cutaneous neurofibromas, and juvenile myelomonocytic leukemia, due to mitotic recombination (copy-neutral loss-of-heterozygosity), microdeletions, or a second variant. Confirmed dual RASopathy diagnoses (most often NF1 plus PTPN11) are rare and produce blended systemic phenotypes. Interpretation of double NF1 findings should prioritize independent American College of Medical Genetics and Genomics/Association for Molecular Pathology classification of each variant, rigorous phasing (segregation/RNA/allele-specific methods), and tumor-focused analysis where applicable. Our index case and synthesis support a practical workflow that clarifies counseling and helps avoid over-calling dual pathogenicity.

Identifiers

PMID42012601
PMCPMC13100077

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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.