Perhaps the most important aspect of therapy for neurodegenerative disease is the early recognition of clinical neurodegeneration and institution of therapy. Studies combining MRI findings together with cerebrospinal fluid markers of demyelination, to identify patients who require therapy, even before onset of clinical symptoms, are currently underway in several countries.
Treatment Options for Childhood LCH No Longer Considered Effective
Treatments that have been used in the past but are no longer recommended for pediatric patients with LCH in any location include cyclosporine  and interferon-alpha. Extensive surgery is also not indicated. Curettage of a circumscribed skull lesion may be sufficient if the lesion in not in the temporal, mastoid, or orbital areas (CNS risk). Patients with disease in these particular sites are recommended to receive 6 months of systemic therapy with vinblastine and prednisone. For lesions of the mandible, extensive surgery may destroy any possibility of secondary tooth development. Surgical resection of groin or genital lesions is contraindicated as these lesions can be healed by chemotherapy.
Radiation therapy use in LCH has been significantly reduced in pediatric patients, and even low-dose radiation therapy should be limited to single-bone vertebral body lesions or other single-bone lesions compressing the spinal cord or optic nerve that do not respond to chemotherapy.
Assessment of Response to Treatment
Response assessment remains one of the most difficult areas in LCH therapy unless there is a specific area that can be followed clinically or with sonography, computed tomography (CT), or MRI scans of areas such as the skin, hepato/splenomegaly, and other mass lesions. Clinical judgment including evaluation of pain and other symptoms remains important.
Bone lesions may take many months to heal and are difficult to evaluate on plain radiographs, although sclerosis around the periphery of a bone lesion suggests healing. CT or MRI scans are useful in assessing response of a soft tissue mass associated with a bone lesion, but is not particularly helpful in lytic bone lesions. Technetium bone scans remain positive in healing bone. Positron emission tomography (PET) scans may be helpful in following the response to therapy since intensity of the PET image diminishes with response of lesions and healing of bone.