Diffuse Intra-Axial expansive Lesion of the Enchephalic Trunk
Short Summary

7-year-old boy presented with general asthenia, hand tremors, anxiety and episodes of diffuse headache. Exam noted facial asymmetry and symptoms progressed to vomiting. The patient had ataxia, dyarthria and facial weakness. CT and MRI revealed a expansile lesion of the brainstem and signs of obstructive hydrocephalus and possible leptomeningeal dissemination. Steriods improved clinical symptoms. The patient started an experimental treatment with Nimotuzumab, and Radiation therapy is planned to continue with Nimotuzumab.

Patient's Questions
1) What therapy do you suggest? If you exclude the surgical operation do you share the pharmacologic therapy + diagnosed radiotherapy? Is there the possibility after the diagnosed therapy, that the tumoral mass could decrease allowing later a solving surgical operation?
2) Do his parents know that the radiotherapy is applicable only once in the same point; afterwards does it exist a follow-up therapy for this kind of tumor that can give long-term and good quality life expectancies?
3) Now  the young patient is taking cortisone that involves an excessive increasing in his appetite compared to his real needs. Is it advisable to regulate the amount and/or to opt for particular types and quality of food? Is it necessary to contact a nutritionist?

4) What will the pathology course be? Will the symptomatology be very painful?


5)  What are the centres of excellence for the treatment of such pathologies?
Medical Background

 7 years old, male.
Diagnosis: DIFFUSE INTRA-AXIAL EXPANSIVE LESION OF THE ENCEPHALIC TRUNK NOT SURGICALLY INVESTIGATED.

Medical history: 
First-born child, he has a younger brother of 5 years old who enjoys a good health. Right-handed child. Not findings of allergies to drugs and food intolerances.
Normal spontaneous delivery. Breastfeeding until six months of age. Psychophysical development within norm limits. He got the vaccinations according to law.
Among the exanthematic diseases: sixth disease when he was 1 and a half year; varicella when he was 5.
No surgical operation in the past and no current home therapy.
Case history:
The clinical symptoms began about one and a half month ago with general asthenia, right hand tremors, state of anxiety and episodes of diffuse headache of short duration, with a frequency of about twice a day with spontaneous remission.
Therefore, he carried out a paediatric visit with evidence of a right asymmetry of the mouth. From 10/2008 repeated vomit episodes occurred, associated with slowing down of the ideomotor functions. On 11/2008 the child was, therefore, brought to the emergency room of the Trento Hospital, where a neurological examination was carried out with finding of a right hand side mouth deviation, uncertain walking, tendency to break up and difficulty in the word articulating activity. A brain CT scan was then performed, with evidence of an expansive lesion on the encephalic trunk that had a swollen appearance. A brain magnetic resonance (MRI) with contrast was then performed, as a further in-depth analysis whose medical report is integrally carried:
“Examination performed as a complement of today’s CAT scan without contrast medium.
We confirm the presence of an expansive lesion of the encephalic trunk, widely infiltrating the pons, the mesencephalon (especially at the right slope), the right cerebellar peduncle, and with an anterior esophitic development with the tendency to wrap the basic artery towards the front.
This formation is characterized by a hyper-intensity in the T2 weighted images, and a hypo-intensity in the T1 weighted images, with nearly absent contrastographic impregnation after Gadolinium (6 ml I.V.) administration: the finding lets hypothesize for a glioma of the encephalic trunk. Nodular enhancement foci are present at clivus level, especially at the right hand side of the median line, likely to be a leptomeningeal dissemination.
Light hyper-intensity of the white peri-ventricular substance close to the frontal and occipital horns in the FLAIR sequence due to an initial subependymal liquor overflow, sign of obstructive hydrocephalus.”
The young patient was, therefore, sent to the Hospital Istituti Ospitalieri of Verona for action.
During this hospitalization it was attempted to carry out a high-field MR imaging (3T), diffusion and spectroscopy, but the child didn’t cooperate for the required time and the procedure was suspended; the neuroradiologists have confirmed what already expressed in the medical report by the colleagues of the Santa Chiara Hospital of Trento, the lesion is, in all probability, a glioma b.g.
For its characteristics of diffuse inherent lesion it is not susceptible of surgery treatment. His parents have consulted a second doctor who has confirmed what it had already been expressed by the colleagues of Trento.
The steroid therapy (Decadron 2 mg x 2 I.V.) has brought about an improvement in the clinical situation at admission.
He was discharged from Verona Hospital on 11/2008 and continued home the current intravenous steroid therapy. Afterwards, on 11/2008, further hospitalization at the National Cancer Institute of Milan, where he is still hospitalized.
With regard to the diagnosed therapy the following elements are reported:
- As for the therapy prescribed to the patient: since 11/08 (date of hospitalization at the Borgo Trento Hospital of Verona, where he remained for three days) the therapy consists of:
Soldesam 0.2% (32 drops in the morning and 16 drops in the afternoon)
Nexium (1 tablet of 20 mg)
- Afterwards, he was moved to the National Cancer Institute of Milan, since 11/08 to the aforesaid drugs the antibody NIMOTUZUMAB (once a week for 12 consecutive times) was added.
The second dosage of NIMOTUZUMAB will be administered on 11/08.

 

Expert's Opinion

Recommendations:
I have reviewed the notes provided and based on the diagnosis of brainstem glioma based on imaging, I would recommend the following to further define the diagnosis and treatment plan:
Answers to Questions:
1) What therapy do you suggest? If you exclude the surgical operation do you share the pharmacologic therapy + diagnosed radiotherapy? Is there the possibility after the diagnosed therapy, that the tumoral mass could decrease allowing later a solving surgical operation?
-       I have not seen the MRI but if there is any exophytic component that can be biopsied that maybe reasonable unless the imaging is completely characteristic. So assuming that other diseases have been ruled out (infection, inflammation, etc) and that this does represent a brainstem glioma I would recommend the following further work-up. The other reason to consider biopsy is that we can obtain molecular signature of tumors even on small sample size which may help in treatment planning in future. However by report there is evidence of leptomeningeal dissemination but I do not see any CSF evaluation- so this would be an important stem to document the type of cells, characterize molecularly and follow for response assessment.

-       An MRI spine to make sure not other lesions in craniospinal axis.

-       From the MRI report the patient has hydrocephalus and so carefully clinical monitoring should be done as some of the patient’s symptoms maybe due to hydrocephalus and this may get worse during radiation when there is more swelling. The patient need a ventriculoperitoneal shunt at some point soon. If this is done, cerebrospinal fluid should be sent for analysis for malignant cells and to rule out other conditions.

-       After surgery is decided and radiation is done then then medical treatments should be considered.

-       The standard treatments for this condition have poor results and so doing more aggressive, clinical trials is a very reasonable treatment plan. And you are doing exactly that with NIMOTUZUMAB. I would stay with this plan for now and then follow with MRIs and stay on this treatment as long as the tumor responds.

-       If there is failure in future then can consider other clinical trials at local hospitals or consider other locations in Europe or USA. 

-       Other more aggressive investigational chemotherapies include temozolomide, erlotonib+rapamycin, bevacizumab + CPT-11, and may other common regimens can also be considered.

2)         Do his parents know that the radiotherapy is applicable only once in the same point; afterwards does it exist a follow-up therapy for this kind of tumor that can give long-term and good quality life expectancies?
-       Radiation can only be given one usually and other options after radiation are discussed above.

3)         Now the young patient is taking cortisone that involves an excessive increasing in his appetite compared to his real needs. Is it advisable to regulate the amount and/or to opt for particular types and quality of food? Is it necessary to contact a nutritionist?
-       The physicians have to decide if the decadron is helping edema due to tumor versus hydrocephalus so that decadron can be tapered over time to reduce these side effects. Yes controlling the type of food intake will help with side effects over time. The other option is consider steroid saving agents such as celecoxib or bevacizumab which would be a more aggressive path.

4)         What will the pathology course be? Will the symptomatology be very painful?
-       If surgical biopsy is decided then it should be fairly safe in the right hands. However, there are risks to every procedure so this should be discussed in detail with a well known pediatric neurosurgeon.

5)         What are the centres of excellence for the treatment of such pathologies in Italy and Europe?
-       I have no personal experience with centers in Europe, but will investigate and respond in time.
 
All of these recommendations have to be taken in context of functional status and balancing quality of life.

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