Langerhans Cell Histiocytosis Medication

Updated: Nov 02, 2022
  • Author: Christopher R Shea, MD; Chief Editor: William D James, MD  more...
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Medication Summary

The goals of pharmacotherapy for Langerhans cell histiocytosis (LCH) are to reduce morbidity and to prevent complications.



Class Summary

These agents have anti-inflammatory properties and cause profound and varied metabolic effects. Corticosteroids modify the body's immune response to diverse stimuli.

Prednisone (Deltasone, Liquid Pred, Meticorten, Orasone, Prednicen-M, Sterapred)

Prednisone is also known as deltacortisone and deltadehydrocortisone. It may decrease inflammation by reversing increased capillary permeability and suppressing PMN activity.

Use the lowest effective dose in elderly patients. Pediatric dosing depends on the condition being treated and the response of the patient; the dose for infants and children should be based on severity and the response of the patient rather than on strict adherence to the dose indicated by age, weight, or body surface area.

Prednisolone (AK-Pred, Articulose-50, Delta-Cortef, Econopred, Inflamase)

Prednisolone is also known as delta hydrocortisone, metacortandralone, prednisolone acetate, and prednisolone sodium phosphate. It decreases inflammation by suppressing the migration of polymorphonuclear leukocytes and reducing capillary permeability.

Methylprednisolone (Adlone, A-methaPred, depMedalone, Depoject, Depopred)

Methylprednisolone is also known as 6-alpha-methylprednisolone, methylprednisolone acetate, and methylprednisolone sodium succinate. By reversing increased capillary permeability and suppressing PMN activity, it may decrease inflammation.


Nonsteroidal anti-inflammatory drugs

Class Summary

These agents are the most commonly used medications to control mild to moderate pain and to decrease inflammation.

Indomethacin (Indocin)

Indomethacin is rapidly absorbed; metabolism occurs in the liver by demethylation, deacetylation, and glucuronide conjugation; it inhibits prostaglandin synthesis.


Antineoplastic agents

Class Summary

These agents inhibit cell growth and proliferation.

Mechlorethamine (Mustargen)

Mechlorethamine forms interstrand and intrastrand cross-links in DNA, which, in turn, results in miscoding, breakage, and failure of replication, inhibiting cell growth. It is dispensed as either an aqueous solution or an ointment. The contents of a 10-mg vial are rehydrated with 50 mL of tap water. The patient should wear protective plastic gloves while applying the solution. Unused preparation may be stored in refrigerator.

Vinblastine (Alkaban-AQ, Velban)

Vinblastine inhibits microtubule formation, which, in turn, disrupts the formation of a mitotic spindle, causing cell proliferation to arrest at metaphase.

Etoposide (Toposar, VePesid)

Etoposide inhibits topoisomerase II and causes DNA strand breakage, causing cell proliferation to arrest in the late S or early G2 portion of the cell cycle.

Mercaptopurine (Purinethol)

Mercaptopurine is a purine analog that inhibits DNA and RNA synthesis, causing cell proliferation to arrest.

Methotrexate (Folex, Rheumatrex)

Methotrexate is an antimetabolite that inhibits DNA synthesis and cell reproduction in malignant cells. Adjust the dose gradually to attain a satisfactory response. Refer to individual protocols; it may be administered through various routes.

2-CdA/Cladribine (Leustatin)

2-CdA/cladribine is a synthetic antineoplastic agent for continuous intravenous infusion. The enzyme deoxycytidine kinase phosphorylates this compound into active 5+-triphosphate derivative, which, in turn, breaks DNA strands and inhibits DNA synthesis. It disrupts cell metabolism, causing death to resting and dividing cells.

Cytarabine (Cytosar-U, Tarabine PFS)

Cytarabine is converted intracellularly to the active compound cytarabine-5'-triphosphate, which inhibits DNA polymerase. It is cell-cycle S-phase specific. Cytarabine blocks the progression from G1 to the S phase and, in turn, kills cells that undergo DNA synthesis in the S phase of the cell proliferation cycle.

Vemurafenib (Zelboraf)

Vemurafenib is an inhibitor of some mutated forms of BRAF serine-threonine kinase, including BRAF-V600. It is indicated for Erdheim-Chester disease (ECD) with BRAF V600 mutation. This is the first FDA-approved treatment for ECD.

Cobimetinib (Cotellic)

Indicated for adults with histiocytic neoplasms eg, (Langerhans cell histiocytosis, Roasi-Dorfman, Erdheim-Chester disease). It is a reversible inhibitor of mitogen-activated protein kinase (MAPK)/extracellular signal regulated kinase 1 (MEK1) and MEK2. MEK proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway, which promotes cellular proliferation. 


Phototherapy agents

Class Summary

PUVA has been a successful therapy for some patients. The goal of treatment is to induce remission of skin diseases by repeated and controlled phototoxic reaction. Photoconjugation of psoralens with DNA produces an antiproliferative reaction in the skin, generates programmed cell death (apoptosis), and induces down-regulation of the cutaneous immune system.

Methoxsalen (8-MOP, Oxsoralen, Oxsoralen Ultra)

Methoxsalen inhibits mitosis by binding covalently to pyrimidine bases in DNA when photoactivated by UV-A. Doses are based on lean body weight.


Bisphosphonate derivatives

Class Summary

These agents inhibit bone resorption by osteoclasts and in turn mitigate associated bone pain.

Zoledronate (Zometa)

Zoledronate is used to treat multiple myeloma and solid tumor bone metastases. It is also used for hypercalcemia of malignancy. It inhibits bone resorption, possibly by acting on osteoclasts or osteoclast precursors. The median duration of complete response (maintaining normalized calcium levels) and time to relapse is reported as 32 and 30 days, respectively. Administer daily calcium and vitamin D when used for multiple myeloma or metastatic bone disease.