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]

Overview

Observation is recommended for all pediatric patients with isolated skin involvement of Langerhans cell histiocytosis. Medical therapy is suggested only for symptomatic cutaneous 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, nitrogen mustard, and oral thalidomide. Curettage is the mainstay treatment for single skull lesions that involve the frontal, parietal, or occipital bones. The most commonly used systemic chemotherapy regimen for the management of multiple bone lesions is a combination of vinblastine and prednisone. The standard therapy length recommended for Langerhans cell histiocytosis involving the spleen, liver, or bone marrow (high-risk organs) consists of vinblastine AND prednisone AND 6-mercaptopurine for a 12 month period.

Treatment

Low-risk Disease (Single-system Unifocal Disease)

Isolated Skin Involvement

• Observation is recommended for all pediatric patients with isolated skin involvement of Langerhans cell histiocytosis.
• Medical therapy is suggested only for symptomatic cutaneous 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 for the management of multiple bone lesions 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 (High-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 Langerhans cell histiocytosis involving the spleen, liver, or bone marrow (high-risk organs) consists of vinblastine AND prednisone AND 6-mercaptopurine for a 12 month period.

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