The purposes of this study are to uncover- Which clinical and neuroradiological measures can be used to predict the (neuropsychological) outcome and progression of symptoms in children with CMS?We hope that the results will contribute to an overall…
ID
Source
Brief title
Condition
- Nervous system neoplasms malignant and unspecified NEC
- Nervous system, skull and spine therapeutic procedures
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary aim is to investigate early clinical and neuroradiological
predictors of neuropsychological outcome in children with posterior fossa
tumors.
Hypothesis: clinical criteria, CMS symptom severity, and changes
inneuroradiological findings over time will predict neuropsychological outcome
at 12 months after surgery. For example, youngerage, greater CMS symptom
severity, and larger changes in white matter metrics (DTI) and cerebral
perfusion (ASL) over time will be associated with poorer neuropsychological
outcomes.
Secondary outcome
Secundary objectives are to identify group differences and changes over time in
neuroradiological and neuropsychological measures for children who have higher
versus lower CMS symptom severity.
Hypothesis: patients with higher versus lower CMS symptom severity will have
greater pre- and post-surgery damage to the efferent cerebellar pathways (DTI),
leading to cerebello-cerebral diaschisis and supratentorial hypoperfusion
(ASL). We expect that some neuroradiological deficits will be shown at the
pre-surgery phase, but impairments will become more apparent post-surgery. Some
children will show recovery over time; however, those with greater CMS symptoms
will have slower recovery.
Background summary
Central nervous system (CNS) tumours constitute 25% of all childhood cancers,
and more than half of these are located in the cerebellum. One of the most
troublesome late effects after operation for such a tumour is the cerebellar
mutism syndrome (CMS) which is seen in up to 25% of children after surgery. It
is characterized by mutism, hypotonia, ataxia and irritability, and the exact
causes have yet to be identified. Although a cure may have been achieved with
respect to their brain tumour, the CMS and its consequences can still represent
a lifelong challenge for these children. Since roughly half of all paediatric
brain tumours reside in the posterior fossa and require operative removal, the
CMS constitutes both a common and severe problem in paediatric neurooncology.
Study objective
The purposes of this study are to uncover
- Which clinical and neuroradiological measures can be used to predict the
(neuropsychological) outcome and progression of symptoms in children with CMS?
We hope that the results will contribute to an overall reduction in incidence
and severity of the CMS as well as increasing
understanding and awareness of the syndrome. Furthermore, this study can lead
to harmonization of the treatment of these
patients.
Study design
Multicenter prospective observational cohort study
Study burden and risks
Patients participating in the study will be treated according to local
standards. Additionally, we will conduct neuropsychological tests
preoperatively, postoperatively, and 12 months after surgery using digital
tests that take approximately 30 minutes each time. While the child completes
the neuropsychological tests under supervision, the parent(s) will be asked to
digitally complete several questionnaires. Furthermore, the child will undergo
an MRI scan as part of standard care. The preoperative, postoperative, and
12-month follow-up MRI scans will also be used for the study. Only 12 months
after the operation, 2 additional MRI sequences will be added to the clinical
protocol, extending the MRI duration by 5-15 minutes.
Participation in the study will not impact or interfere with the child's
treatment plan. The child will not experience any additional benefit or risk.
Cerebellar Mutism Syndrome (CMS) predominantly occurs in children. There are
few case reports of CMS in adults in the literature. Therefore, it would not be
possible to conduct this study with adult patients.
Besides contributing to an increased understanding and awareness of the CMS
this study has the direct prospect of reducing both the incidence and the
severity of the syndrome, which would be a great advantage for other children
with CMS.
Heidelberglaan 25
Utrecht 3584 CS
NL
Heidelberglaan 25
Utrecht 3584 CS
NL
Listed location countries
Age
Inclusion criteria
- Eligible for European CMS study
- Age 2-18 years at the date of first imaging showing this tumour
- Understanding and speaking of Dutch language by patient and/or parents
Inclusion criteria European CMS study:
- Age <18 years at the date of first imaging showing this tumour
- Tumour in the cerebellum/4th ventricle/brainstem with intention to treat with
surgical resection or open biopsy.
- Signed Informed consent from custodial parent(s) and/or patient
Exclusion criteria
- Patients who have had previous surgery of the posterior fossa
- No informed consent
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| ISRCTN | ISRCTNnumbertobeconfirmed |
| CCMO | NL85971.041.23 |