Langerhans cell histiocytosis medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Haytham Allaham, M.D. [2]

Treatment

Treatment of Low-risk Disease (Single-system or Multisystem)

Isolated Skin Involvement

• Observation is recommended for all pediatric patients with isolated skin involvement of Langerhans cell histiocytosis.
• Therapy is suggested only for symptomatic disease that presents with either extensive rash, sever pain, skin ulceration, or bleeding.
• Medical therapies used for the treatment of isolated skin lesions of Langerhans cell histiocytosis include:

• Topical steroids
• Oral methotrexate (20 mg/m2) weekly for 6 months
• Oral thalidomide 50 mg to 200 mg each night
• Topical application of nitrogen mustard can be effective for cutaneous Langerhans cell histiocytosis that is resistant to oral therapies, but not for disease involving large areas of skin.
• Psoralen and long-wave ultraviolet A radiation (PUVA) and UVB can be effective in skin Langerhans cell histiocytosis but its use is limited by the potential for late skin cancers.

Skull Involvement

• Curettage is the mainstay treatment for single skull lesions that involve the frontal, parietal, or occipital bones.
• In certain cases, curettage plus an injection of methylprednisolone may be used
• The use of low-dose radiation therapy is limited among pediatric patients due to severe side effects.
• Patients with isolated lesions to the mastoid, temporal, or orbital bones are treated with systemic therapy, as to prevent the development of diabetes insipidus among such patients.
• Comparison of diabetes insipidus incidence with no systemic therapy (40%) versus 6 months of vinblastine/prednisone (20%) strongly supports systemic treatment of of such bony lesions.

Vertebral or Femoral Bone Lesions at Risk for Collapse

• Observation is recommended for the management of a single vertebral body lesion that has no soft tissue extension into the extradural space.
• Low-dose radiation therapy may be used to try to promote resolution in an isolated vertebral body lesion or a large femoral neck lesion at risk of fracture
• Despite the low dose required (700–1,000 cGy), radiation therapy should be used with caution in the area of the thyroid gland, brain, or any growth plates.
• Patients with soft tissue extension from vertebral lesions are often treated successfully with chemotherapy.

Multiple Bone Lesions (Single-system Multifocal Bone Disease)

• The most commonly used systemic chemotherapy regimen is a combination of vinblastine and prednisone.
• Vinblastine treatment is recomended for 12 months. Weekly doses for the first 7 weeks, which is followed by a dose every 3 weeks for the rest of the management period.
• Prednisone (40 mg/m2) is given daily for 4 weeks then tapered over 2 weeks. Afterwards prednisone is given for 5 days at 40 mg/m2 every 3 weeks with the vinblastine injections.

Multiple Bone Lesions in Combination with Skin, Lymph Node, or Diabetes Insipidus (Low-risk Multisystem Disease)

• Vinblastine AND prednisone combination therapy is the mainstay treatment for low-risk multisystem Langerhans cell histiocytosis.
• The same chemotherapy regimen of vinblastine and prednisone as described above is used for 12 months.
• Other chemotherapeutic agents used for the treatment of low-risk multisystem Langerhans cell histiocytosis include:

• Vincristine AND cytosine arabinoside AND prednisone
• Cladribine
• Pamidronate
• Zoledronate
• Alendronate

Treatment of High-risk Multisystem Disease

• The standard therapy length recommended for LCH involving the spleen, liver, or bone marrow (high-risk organs) is now 12 months
• The mainstay therapy consists of vinblastine AND prednisone AND 6-mercaptopurine

Treatment of CNS Langerhans cell histiocytosis

• There are three types of Langerhans cell histiocytosis CNS lesions:

• Mass lesions or tumors in the cerebrum, cerebellum, or choroid plexus
• Mass lesions of the hypothalamic-pituitary axis that are always associated with diabetes insipidus
• Neurodegenerative syndrome

• Drugs that cross the blood-brain barrier, such as cladribine, or other nucleoside analogs, such as cytarabine, are used for active CNS Langerhans cell histiocytosislesions.
• Immunochemotherapeutic agents used for the treatment of Langerhans cell histiocytosis neurodegenerative syndrome include:

• Dexamethasone
• Cladribine
• Retinoic acid
• Intravenous immunoglobulin (IVIg)
• Infliximab
• Cytarabine
• Vincristine