ALK-rearranged histiocytosis: Report of two cases with involvement of the central nervous system.


Journal

Neuropathology and applied neurobiology
ISSN: 1365-2990
Titre abrégé: Neuropathol Appl Neurobiol
Pays: England
ID NLM: 7609829

Informations de publication

Date de publication:
10 2021
Historique:
revised: 15 05 2021
received: 10 02 2021
accepted: 24 05 2021
pubmed: 29 5 2021
medline: 29 1 2022
entrez: 28 5 2021
Statut: ppublish

Résumé

Histiocytoses are a heterogeneous group of localized or disseminated diseases. Clinical presentation and patients' outcome vary greatly, ranging from mild to life-threatening disorders. Rare cases of systemic or localized histiocytosis harboring ALK rearrangement have been reported. Two cases of CNS histiocytosis were thoroughly investigated by implementing multiple molecular tests, i.e. FISH, RT-qPCR, NGS analysis. In a 10-month old girl (patient #1), MRI showed two left hemispheric lesions and a right fronto-mesial lesion histologically consisting of a moderately cellular infiltrative proliferation, composed by CD68(PGM1)+/CD163+ spindle cells. ALK 5'/3'-imbalance and a KIF5B(exon 24)-ALK(exon 20) fusion were documented by RT-qPCR and NGS analysis, respectively. A subsequent CT scan showed multiple hepatic and pulmonary lesions. The patient was started on chemotherapy (vinblastine) associated to an ALK-inhibitor (Alectinib) with remarkable response. In a 11-year-old girl (patient #2), MRI showed a right frontal 1.5 cm lesion. Neuropathological examination revealed a histiocytic proliferation composed by medium sized CD68(PGM1)+/HLA-DR+ cells, showing moderate ALK1 positivity. ALK rearrangement and a KIF5B(exon 24)-ALK(exon 20) fusion were demonstrated also in this case. Subsequent CT, 18F-FDG-PET and MRI scans showed the presence of a single right femoral lesion, proved to be a fibrous cortical defect. In ALK-histiocytoses, CNS involvement may occur as part of a systemic disease or, rarely, as its only primary disease localization, which could remain otherwise asymptomatic. The diagnosis often relies on neuropathological examination of brain biopsy, which may pose a diagnostic challenge due to the variable histopathological features. An integrated histological and molecular approach in such cases is recommended.

Identifiants

pubmed: 34048085
doi: 10.1111/nan.12739
doi:

Substances chimiques

Protein Kinase Inhibitors 0
ALK protein, human EC 2.7.10.1
Anaplastic Lymphoma Kinase EC 2.7.10.1
Receptor Protein-Tyrosine Kinases EC 2.7.10.1

Types de publication

Case Reports Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

878-881

Informations de copyright

© 2021 British Neuropathological Society.

Références

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Auteurs

Sabrina Rossi (S)

Pathology Unit, Department of Laboratories, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Marco Gessi (M)

Neuropathology Unit, Pathology Division, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Sabina Barresi (S)

Pathology Unit, Department of Laboratories, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Gianpiero Tamburrini (G)

Pediatric Neurosurgery Unit, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Isabella Giovannoni (I)

Pathology Unit, Department of Laboratories, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Antonio Ruggiero (A)

Pediatric Oncology Division, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Giovanna Stefania Colafati (GS)

Neuroradiology Unit, Imaging Department, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Paolo Frassanito (P)

Pediatric Neurosurgery Unit, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Alessia Carboni (A)

Neuroradiology Unit, Imaging Department, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Andrea Alexandre (A)

Radiology and Neuroradiology Division, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Antonella Cacchione (A)

Department of Onco-Hematology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Pietro Trombatore (P)

Radiology and Neuroradiology Division, Fondazione Policlinico Universitario A.Gemelli IRCCS, Università Cattolica S.Cuore, Rome, Italy.

Francesca Diomedi-Camassei (F)

Pathology Unit, Department of Laboratories, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Stefania Gaspari (S)

Department of Onco-Hematology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Francesca Gianno (F)

Department of Radiology, Oncology and Anatomic Pathology, University La Sapienza, Rome, Italy.
IRCCS Neuromed, Pozzilli, Isernia, Italy.

Carlo Efisio Marras (CE)

Neurosurgery Unit, Department of Neuroscience and Neurorehabilitation, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Valerio Cecinati (V)

Pediatrics and Pediatric Oncoematology Unit, Ospedale Santissima Anninziata, Taranto, Italy.

Andrea Carai (A)

Neurosurgery Unit, Department of Neuroscience and Neurorehabilitation, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Angela Mastronuzzi (A)

Department of Onco-Hematology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Rita Alaggio (R)

Pathology Unit, Department of Laboratories, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

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