Skip to content

Cancer Health Center

Font Size

Treatment of Childhood LCH


Treatment of CNS disease

Although CNS LCH arises initially at areas where the blood brain barrier is deficient, drugs that cross the blood-brain barrier, such as cladribine (2-CdA), or other nucleoside analogs, such as cytarabine, seem to be the best option for active CNS LCH lesions.

  • Treatment of mass lesions with cladribine (2-CdA) has been effective in 13 reported cases.[26,27]; [28][Level of evidence: 3iiiDiii] Mass lesions included enlargement of the hypothalamic-pituitary axis, parenchymal mass lesions, and leptomeningeal involvement. Doses of 2-CdA ranged from 5 mg/m2 to 13 mg/m2 given at varying frequencies.[28][Level of evidence: 3iiiDiii]
  • LCH patients with mass lesions in the hypothalamic-pituitary region, the choroid plexus, the grey matter, or the white matter may respond to chemotherapy.[29,30] Treatment with vinblastine with or without corticosteroids for patients with CNS mass lesions (20 patients; mainly pituitary) demonstrated an objective response in 15 patients, with 5 of 20 patients demonstrating a complete response and 10 of 20 patients demonstrating a partial response.
  • For treatment of symptoms of LCH CNS neurodegenerative syndrome, dexamethasone, 2-CdA, retinoic acid, intravenous immunoglobulin (IVIg), infliximab, and cytarabine with or without vincristine have been used.[28][Level of evidence: 3iiiDiii]; [28,31,32,33,34,35] Retinoic acid was given at a dose of 45 mg/m2 daily for 6 weeks, then 2 weeks per month for 1 year.[31] IVIg (400 mg/m2) was given monthly and chemotherapy consisting of oral prednisolone with or without oral or intravenous methotrexate and oral 6-mercaptopurine were given for at least 1 year.[32] Magnetic resonance imaging (MRI) findings were stable but clinical efficacy was difficult to judge as patients were reported to have no progression in their neurologic symptoms. A study using cytarabine with or without vincristine reported improvement in the clinical and MRI findings.[34][Level of evidence: 3iiiC] Seven of eight patients have been followed for more than 2 years after stopping therapy and have had stable neurologic and radiographic findings.

    In the Japan LCH Study Group-96 Protocol study patients received cytarabine 100 mg/m2 daily on days 1 to 5 during induction and 150 mg/m2 on day 1 of each maintenance cycle (every 2 weeks for 6 months). Three of 91 patients developed neurodegenerative disease, which is similar to the experience on Histiocyte Society studies.[25]

Reactivation of single-system and multisystem LCH

Reactivation of LCH after complete response has been reported; usually occurring within the first 9 to 12 months after stopping treatment.[36] The percentage of patients with reactivations was 17.4% for single-site disease; 37% for single-system, multifocal disease; 46% for multisystem (nonrisk organ) disease; and 54% for patients with risk-organ involvement. Forty-three percent of reactivations were in bone, 11% in ears, 9% in skin, and 7% develop diabetes insipidus; a lower percentage of patients had lymph node, bone marrow, or risk-organ relapses.[36] The median time to reactivation was 12 to 15 months in nonrisk patients and 9 months in risk patients. One-third of patients had more than one reactivation varying from 9 to 14 months after the initial reactivation. Patients with reactivations were more likely to have long-term sequelae in the bones, diabetes insipidus, or other endocrine, ear, or lung problems.[36]


Today on WebMD

Colorectal cancer cells
A common one in both men and women.
Lung cancer xray
See it in pictures, plus read the facts.
sauteed cherry tomatoes
Fight cancer one plate at a time.
Ovarian cancer illustration
Do you know the symptoms?
Jennifer Goodman Linn self-portrait
what is your cancer risk
colorectal cancer treatment advances
breast cancer overview slideshow
prostate cancer overview
lung cancer overview slideshow
ovarian cancer overview slideshow
Actor Michael Douglas