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Disease

Histiocytosis

Late-stage therapeutic developmentEmerging research
MeSHHistiocytosis
2
Publications
18
Clinical trials
2022
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q1 2027.
Upcoming Milestones
Clinical Milestones6View
Results expected 4
Recently completed 2
Activity timeline6

Clinical trials

13 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
18
All trials
6
Active
4
Late-stage
6
Completed
Late-stage studies

Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)

Phase 3 · Recruiting · Baylor College of Medicine

Determination of Molecular Status, as Well as the Efficacy and Safety of Fluorodeoxyglucose (18F-FDG) in PET-CT Imaging in Juvenile Patients With Histiocytosis

Phase 3 · Recruiting · Anna Raciborska

LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis

Phase 2/3 · Active, not recruiting · North American Consortium for Histiocytosis

In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders

Phase 2/3 · Terminated · Masonic Cancer Center, University of Minnesota
Recruiting

Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)

Phase 3 · Recruiting · Baylor College of Medicine

Determination of Molecular Status, as Well as the Efficacy and Safety of Fluorodeoxyglucose (18F-FDG) in PET-CT Imaging in Juvenile Patients With Histiocytosis

Phase 3 · Recruiting · Anna Raciborska

A Phase 2 Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders.

Phase 2 · Recruiting · Carl Allen

Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis

Phase 2 · Recruiting · National Cancer Institute (NCI)

Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib

Phase 2 · Recruiting · Cook Children's Health Care System
Recently completed

Evaluation of Efficacy of Denosumab in Adult Patients With Langerhans Cell Histiocytosis (LCH): a Multiple-site, Single Arm, Open Label Clinical Trial

Phase 2 · Completed · Hellenic Society for the Study of Bone Metabolism

Efficacy and Tolerance of Combination Chemotherapy With Methotrexate and Cytosine Arabinoside in Newly Diagnosed Adult With Langerhans Cell Histiocytosis

Phase 2 · Completed · Peking Union Medical College Hospital

A Phase 2a, Open Label, Multicenter Study to Assess the Efficacy and Safety of the Oral AKT Inhibitor GSK2110183 in Subjects With Langerhans Cell Histiocytosis

Phase 2 · Completed · GlaxoSmithKline

Treatment Protocol of the Third International Study For Langerhans Cell Histiocytosis

N/A · Completed · Histiocyte Society

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Phase 2 · Completed · Masonic Cancer Center, University of Minnesota

Treatment of Resistant Langerhans Cell Histiocytosis With Etanercept (ENBREL, IMMUNEX, SEATTLE)

Phase 2 · Completed · Baylor College of Medicine

Research activity

2 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20152022
Major themes3
  • Hematologic Neoplasms1
  • Histiocytosis1
  • Mutation1
Leading journals2
  • Leukemia1
  • The New England journal of medicine1
Leading researchers8
  • Abla O1
  • Akkari Y1
  • Alaggio R1
  • Apperley JF1
  • Baselga J1
  • Bejar R1
  • Berti E1
  • Blay JY1
Affiliations (unnormalised)6
  • Aichi Medical University Hospital1
  • Ambroise Pare Hospital1
  • and Clinical Immunology1
  • Baylor College of Medicine1
  • Beth Israel Deaconess Medical Center1
  • Centre for Haematology1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

General term for the abnormal appearance of histiocytes in the blood. Based on the pathological features of the cells involved rather than on clinical findings, the histiocytic diseases are subdivided into three groups: HISTIOCYTOSIS, LANGERHANS CELL; HISTIOCYTOSIS, NON-LANGERHANS-CELL; and HISTIOCYTIC DISORDERS, MALIGNANT.

Identifiers
MeSHHistiocytosis
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.