HEALTH PROFESSIONAL · SOURCE READING
Treatment options under clinical evaluation
Source: Langerhans Cell Histiocytosis Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: January 6, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
Information about National Cancer Institute (NCI)–supported clinical trials can be found on the NCI website. For information about clinical trials sponsored by other organizations, see the ClinicalTrials.gov website.
Source links and citations
The following are examples of national and/or institutional clinical trials that are currently being conducted:
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Source links and citations
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum I: First-line treatment for multisystem LCH patients (group 1) and patients with single-system LCH with multifocal bone or CNS-risk lesions (group 2).
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum II: Second-line treatment for non–risk-organ patients (patients without risk-organ involvement who fail first-line therapy or have a reactivation after completion of first-line therapy).
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum III: Salvage treatment for risk-organ LCH (patients with dysfunction of risk organs who fail first-line therapy).
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum IV: Hematopoietic stem cell transplant for risk-organ LCH (patients with dysfunction of risk organs who fail first-line therapy).
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum V: Monitoring and treatment of isolated tumorous and neurodegenerative CNS LCH.
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum VI: Natural history and management of other single-system LCH (patients who do not need systemic therapy at the time of diagnosis).
HISTSOC-LCH-IV (NCT02205762) (LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With LCH): On the basis of features at presentation and response to treatment, the LCH-IV study tailors treatment to one of the following seven strata:
Stratum VII (long-term follow up): All patients, regardless of previous therapy, will be monitored for reactivation or permanent consequences once complete disease resolution has been achieved and the respective protocol treatment has been completed.
NCT02670707 (Cytarabine or Vinblastine Sulfate and Prednisone in Treating Patients With LCH): The purpose of this trial is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH.
Source links and citations
It is preferable that patients with LCH be enrolled in a clinical trial whenever possible so that advances in therapy can be achieved more quickly, using evidence-based recommendations, and to ensure optimal care. Information about clinical trials for LCH in children is available from the NCI website, Histiocyte Society website, and the North American Consortium for Histiocytosis (NACHO) website.
Source links and citations
Studies cited in this source section
Source citation is not a determination that a study applies to you.
Preserved source evidence · Not medical advice
Triangle