The frequent and clinically benign anomalies of chromosomes 7 and 20 in Shwachman-diamond syndrome may be subject to further clonal variations.

Bone marrow rescue Chromosome anomalies Karyotype instability Shwachman-Diamond syndrome

Journal

Molecular cytogenetics
ISSN: 1755-8166
Titre abrégé: Mol Cytogenet
Pays: England
ID NLM: 101317942

Informations de publication

Date de publication:
24 Nov 2021
Historique:
received: 31 08 2021
accepted: 03 11 2021
entrez: 25 11 2021
pubmed: 26 11 2021
medline: 26 11 2021
Statut: epublish

Résumé

An isochromosome of the long arm of chromosome 7, i(7)(q10), and an interstitial deletion of the long arm of chromosome 20, del(20)(q), are the most frequent anomalies in the bone marrow of patients with Shwachman-Diamond syndrome, which is caused in most cases by mutations of the SBDS gene. These clonal changes imply milder haematological symptoms and lower risk of myelodysplastic syndromes and acute myeloid leukaemia, thanks to already postulated rescue mechanisms. Bone marrow from fourteen patients exhibiting either the i(7)(q10) or the del(20)(q) and coming from two large cohorts of patients, were subjected to chromosome analyses, Fluorescent In Situ Hybridization with informative probes and array-Comparative Genomic Hybridization. One patient with the i(7)(q10) showed a subsequent clonal rearrangement of the normal chromosome 7 across years. Four patients carrying the del(20)(q) evolved further different del(20)(q) independent clones, within a single bone marrow sample, or across sequential samples. One patient with the del(20)(q), developed a parallel different clone with a duplication of chromosome 3 long arm. Eight patients bore the del(20)(q) as the sole chromosomal abnormality. An overall overview of patients with the del(20)(q), also including cases already reported, confirmed that all the deletions were interstitial. The loss of material varied from 1.7 to 26.9 Mb and resulted in the loss of the EIF6 gene in all patients. Although the i(7)(q) and the del(20)(q) clones are frequent and clinically benign in Shwachman Diamond-syndrome, in the present work we show that they may rearrange, may be lost and then reconstructed de novo, or may evolve with independent clones across years. These findings unravel a striking selective pressure exerted by SBDS deficiency driving to karyotype instability and to specific clonal abnormalities.

Sections du résumé

BACKGROUND BACKGROUND
An isochromosome of the long arm of chromosome 7, i(7)(q10), and an interstitial deletion of the long arm of chromosome 20, del(20)(q), are the most frequent anomalies in the bone marrow of patients with Shwachman-Diamond syndrome, which is caused in most cases by mutations of the SBDS gene. These clonal changes imply milder haematological symptoms and lower risk of myelodysplastic syndromes and acute myeloid leukaemia, thanks to already postulated rescue mechanisms.
RESULTS RESULTS
Bone marrow from fourteen patients exhibiting either the i(7)(q10) or the del(20)(q) and coming from two large cohorts of patients, were subjected to chromosome analyses, Fluorescent In Situ Hybridization with informative probes and array-Comparative Genomic Hybridization. One patient with the i(7)(q10) showed a subsequent clonal rearrangement of the normal chromosome 7 across years. Four patients carrying the del(20)(q) evolved further different del(20)(q) independent clones, within a single bone marrow sample, or across sequential samples. One patient with the del(20)(q), developed a parallel different clone with a duplication of chromosome 3 long arm. Eight patients bore the del(20)(q) as the sole chromosomal abnormality. An overall overview of patients with the del(20)(q), also including cases already reported, confirmed that all the deletions were interstitial. The loss of material varied from 1.7 to 26.9 Mb and resulted in the loss of the EIF6 gene in all patients.
CONCLUSIONS CONCLUSIONS
Although the i(7)(q) and the del(20)(q) clones are frequent and clinically benign in Shwachman Diamond-syndrome, in the present work we show that they may rearrange, may be lost and then reconstructed de novo, or may evolve with independent clones across years. These findings unravel a striking selective pressure exerted by SBDS deficiency driving to karyotype instability and to specific clonal abnormalities.

Identifiants

pubmed: 34819134
doi: 10.1186/s13039-021-00575-w
pii: 10.1186/s13039-021-00575-w
pmc: PMC8611838
doi:

Types de publication

Journal Article

Langues

eng

Pagination

54

Subventions

Organisme : NIDDK NIH HHS
ID : RC2 DK122533
Pays : United States
Organisme : NIH HHS
ID : 1RC2DK122533
Pays : United States
Organisme : Shwachman Diamond Syndrome Foundation
ID : Grant 2020

Informations de copyright

© 2021. The Author(s).

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Auteurs

Abdul Waheed Khan (AW)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.

Alyssa Kennedy (A)

Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.

Elissa Furutani (E)

Keros Therapeutics, Lexington, MA, USA.

Kasiani Myers (K)

Cincinnati Children's Hospital and Medical Center, Cincinnati, OH, USA.

Annalisa Frattini (A)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.
Istituto di Ricerca Genetica e Biomedica, CNR, Milano, Italy.

Francesco Acquati (F)

Dipartimento di Biotecnologie e Scienze della Vita, Università Dell'Insubria, Varese, Italy.
Centro di Medicina Genomica-Università dell'Insubria, Varese, Italy.

Pamela Roccia (P)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.

Giovanni Micheloni (G)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.

Antonella Minelli (A)

Genetica Medica, Fondazione IRCCS Policlinico S. Matteo and Università di Pavia, Pavia, Italy.

Giovanni Porta (G)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.
Centro di Medicina Genomica-Università dell'Insubria, Varese, Italy.

Marco Cipolli (M)

Centro Fibrosi Cistica, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.

Simone Cesaro (S)

Oncoematologia Pediatrica, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.

Cesare Danesino (C)

Genetica Medica, Fondazione IRCCS Policlinico S. Matteo and Università di Pavia, Pavia, Italy.

Francesco Pasquali (F)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy.

Akiko Shimamura (A)

Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.

Roberto Valli (R)

Genetica Umana e Medica, Dipartimento di Medicina e Chirurgia, Università Dell'Insubria, Via J. H. Dunant, 5, 21100, Varese, Italy. roberto.valli@uninsubria.it.
Centro di Medicina Genomica-Università dell'Insubria, Varese, Italy. roberto.valli@uninsubria.it.

Classifications MeSH