Abstract Details

Title Isolated Nervous System Histiocytic Neoplasms: Clinical Manifestations, Treatment, and Outcomes

Topic Neuro-oncology

Presentation(s) P4 - Poster Session 4 (11:45 AM-12:45 PM)

Poster/Presentation
Number 5-018

Objective To describe the frequency, clinical manifestations, imaging findings, and treatment response in individuals with isolated nervous system histiocytic neoplasms at initial presentation, and to determine the frequency at which those individuals later develop systemic manifestations.

Background Nervous system involvement of histiocytic neoplasms, including Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), and Rosai-Dorfman disease (RDD) has been described in those with systemic manifestations. However, longitudinal studies in those with isolated nervous system involvement at initial presentation are more limited, and thus it is unknown whether those with initially only nervous system involvement differ in their treatment response, and whether treatment may help prevent future systemic involvement.

Design/Methods This retrospective cohort study of adult patients with histiocytic neoplasms took place at a tertiary care referral center. Inclusion criteria were: 1) diagnosis of a histiocytic neoplasm based on nervous system biopsy and appropriate clinical phenotype; 2) lack of systemic involvement based on initial body PET or CT of the chest, abdomen, and pelvis and nuclear bone scam, if available; and 3) sufficient medical records for review.

Results Isolated nervous system involvement at initial presentation was seen in 19/377 (5%) of histiocytic neoplasms: 3% (6/228) with LCH, 7% (7/103) with ECD, and 13% (6/46) with RDD. The most common neurological symptoms and signs were headache (11/19, 58%), weakness (8/19, 42%), and diabetes insipidus (7/19, 37%). Three patients later developed systemic involvement, including 1 with LCH (6 years later), and 2 with ECD (each <12 months later). BRAF V600E mutation was present in 29% (4/14 tested). Treatments were individualized with 50% showing clinical response and 67% showing radiological response ≥3 months after treatment.

Conclusions Isolated nervous system involvement of histiocytic neoplasms at initial presentation is uncommon. Surveillance systemic imaging should be undertaken even after initially being negative, as systemic manifestations can occur later.

Authors/Disclosures
Nabeela Nathoo, MD, PhD PRESENTER	Dr. Nathoo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis.
Joon H. Uhm, MD, FRCP(C), FAAN (Mayo Clinic)	Dr. Uhm has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Zai Lab.
Alyx B. Porter, MD, FAAN (Mayo Clinic)	Dr. Porter has a non-compensated relationship as a Board Member with American Brain Foundation that is relevant to AAN interests or activities.
Julie E. Hammack, MD, FAAN (Mayo Clinic Jacksonville)	Dr. Hammack has nothing to disclose.
Kurt A. Jaeckle, MD, FAAN (Mayo Clinic Florida)	Dr. Jaeckle has stock in Entegrion, Inc.
Maciej M. Mrugala, MD, PhD, MPH, FAAN (Mayo Clinic)	Dr. Mrugala has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Veevo Biomedicines Inc. Dr. Mrugala has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arbor Pharmaceuticals. Dr. Mrugala has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra-Zeneca. Dr. Mrugala has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Servier. Dr. Mrugala has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medscape. Dr. Mrugala has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Mrugala has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyiatec . The institution of Dr. Mrugala has received research support from Arbor Pharmaceuticals. Dr. Mrugala has a non-compensated relationship as a Program Director with Society for Neuro-Oncology that is relevant to AAN interests or activities.
Brian Crum, MD (Mayo Clinic)	Dr. Crum has nothing to disclose.
Eoin P. Flanagan, MBBCh, FAAN (Mayo Clinic)	The institution of Dr. Flanagan has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Flanagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pharmacy times. The institution of Dr. Flanagan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. The institution of Dr. Flanagan has received research support from UCB. The institution of Dr. Flanagan has received research support from Roche. The institution of Dr. Flanagan has received research support from UCB. The institution of Dr. Flanagan has received research support from Merck. The institution of Dr. Flanagan has received research support from Roche. Dr. Flanagan has received publishing royalties from a publication relating to health care. Dr. Flanagan has received publishing royalties from a publication relating to health care. Dr. Flanagan has a non-compensated relationship as a Member of medical Advisory Board with The MOG Project that is relevant to AAN interests or activities. Dr. Flanagan has a non-compensated relationship as a Editorial board member with Journal of The Neurologic Sciences that is relevant to AAN interests or activities. Dr. Flanagan has a non-compensated relationship as a Editorial board member with Neuroimmunology Reports that is relevant to AAN interests or activities. Dr. Flanagan has a non-compensated relationship as a Editorial Board Member with Neurology, Neuroimmunology Neuroinflammation (N2) Journal that is relevant to AAN interests or activities. Dr. Flanagan has a non-compensated relationship as a Editorial Board Member with Neurology that is relevant to AAN interests or activities.
Sean J. Pittock, MD, FAAN (Mayo Clinic Dept of Neurology)	Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arialys. The institution of Dr. Pittock has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Dr. Pittock has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. The institution of Dr. Pittock has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion/AstraZeneka. The institution of Dr. Pittock has received research support from NIH. Dr. Pittock has received intellectual property interests from a discovery or technology relating to health care. Dr. Pittock has received intellectual property interests from a discovery or technology relating to health care. Dr. Pittock has received publishing royalties from a publication relating to health care.
Gaurav Goyal	Gaurav Goyal has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Recordati. Gaurav Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sobi. Gaurav Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Electra.
Jason Young (Mayo)	Jason Young has nothing to disclose.
Matthew Koster	The institution of Matthew Koster has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. The institution of Matthew Koster has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rigel. The institution of Matthew Koster has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. The institution of Matthew Koster has received research support from Sobi.
Robert Vassallo	Robert Vassallo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Regeneron. Robert Vassallo has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Rion. Robert Vassallo has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Robert Vassallo has stock in Rion LLC. The institution of Robert Vassallo has received research support from NIH. The institution of Robert Vassallo has received research support from DOD. Robert Vassallo has received intellectual property interests from a discovery or technology relating to health care.
Jay Ryu (Mayo Clinic)	Jay Ryu has nothing to disclose.
Caroline Davidge-Pitts	No disclosure on file
Corrie Bach	No disclosure on file
Aishwarya Ravindran (Mayo Clinic)	Aishwarya Ravindran has nothing to disclose.
Julio Sartori-Valinotti (Mayo Clinic College of Medicine)	Julio Sartori-Valinotti has nothing to disclose.
N. Nora Bennani (Mayo Clinic)	No disclosure on file
Jithma Abeykoon (Mayo Clinic)	Jithma Abeykoon has nothing to disclose.
Mithun Shah	No disclosure on file
Carl Hook	No disclosure on file
Karen Rech	Karen Rech has nothing to disclose.
Ronald Go	Ronald Go has nothing to disclose.
W. O. Tobin, PhD, MBBCh, BAO, FAAN (Mayo Clinic)	Dr. Tobin has received publishing royalties from a publication relating to health care.

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