International Rare Histiocytic Disorders Registry (IRHDR)

NCT02285582 | Recruiting

• 1 other identifier: 1000045224
• Study Type: observational
• Target: 400
• Locations: 9 countries
• Sites: 15

Brief Summary

The rare histiocytic disorders (RHDs) are characterized by the infiltration of one or more organs by non-LCH histiocytes. They can range from localized disease that resolves spontaneously, to progressive disseminated forms that can be sometimes life-threatening. Since they are extremely rare, there is limited understanding of their causes and best treatment options. Physicians, patients and parents of children with RHDs frequently consult members of the Histiocyte Society regarding the best management of these disorders. Very often, no specific recommendation can be made due to the lack of prospective outcome data, or even large retrospective case series. The creation of an international rare histiocytic disorders registry (IRHDR) could facilitate a uniform diagnosis of the RHDs, as well as the collection and analysis of the clinical, epidemiological, treatment and survival data of patients with RHD. The registry may also lead to future therapeutic recommendations, provide a framework for future clinical trials and create excellent research opportunities.

Conditions

Rare Histiocytic Disorders (RHDs); Erdheim-Chester Disease (ECD); Rosai-Dorfman Disease (RDD); Xanthogranuloma Family (XG); Indeterminate Dendritic Cell Histiocytosis; Malignant Histiocytic Neoplasm (MHN); ALK-positive Histiocytosis; Mixed Histiocytosis (MXH); Multicentric Reticulohistiocytoma (MRH); Necrobiotic Xanthogranuloma (NX)

Outcome Measures

Primary Outcomes (1)

• Collecting data on disease presentation, treatments used and treatment outcomes over time for patients diagnosed with RHD to better understand the diseases and optimize the treatments.

  Collecting data on disease presentation, treatments used and treatment outcomes over time for patients diagnosed with RHD to better understand the diseases and optimize the treatments. Data will be analysed on average yearly, the registry will be ongoing for 10 years.

  Data will be analysed on average yearly, the registry will be ongoing for 10 years.

Secondary Outcomes (1)

• Develop treatment guidelines for the RHD based on solid clinical trial data.

  Data will be analysed on average yearly, the registry will be ongoing for 10 years.

Interventions

Registry study OTHER

No intervention.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Rare Histiocytic Disorders: 1. Xanthogranuloma Family (XG) * Cutaneous * Benign cephalic histiocytosis (BCH) * Generalized eruptive histiocytosis (GEH) * Progressive nodular histiocytosis (PNH) * Xanthoma disseminatum (XD) * Giant XG * Reticulohistiocytoma * Ocular * Systemic 2. Erdheim-Chester Disease (ECD) 3. Rosai-Dorfman disease (RDD) * Single system * Nodal * Extranodal * Skin * CNS * Bone * Orbit * Other * Multisystemic 4. Indeterminate Dendritic Cell Histiocytosis 5. Malignant Histiocytic Neoplasm (MHN) * Histiocytic * Langerhans cell * Interdigitating dendritic cell * Indeterminate dendritic cell * Primary MHN * Secondary to: * ALL * Follicular lymphoma * Other B-cell lymphoma * Other hematologic malignancy * Histiocytosis 6. ALK-positive Histiocytosis 7. Mixed Histiocytosis (MXH) * ECD/LCH * RDD/LCH * RDD/ECD * LCH/JXG * Other 8. Other * Multicentric reticulohistiocytoma (MRH) * Necrobiotic xanthogranuloma (NX) * Not otherwise specified (NOS)

You may qualify if:

• Any age at diagnosis.
• Diagnosis of a rare histiocytic disorder, established before or after the opening of the registry.
• Cases diagnosed from January - 01- 1995 until the present time and prospectively.
• Signed informed consent by a patient, or parent/legal guardian.
• Cognitively impaired patients can be included after consent by legal guardian/parent.
• Deceased patients can be included if they are contacted at least 6 months after the death of their child and not on their child's birthday or anniversary of death.

You may not qualify if:

• Informed consent has not been signed.
• Diagnosis other than RHD.
• Cases diagnosed before the year 1995.

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (15)

The University of Alabama at Birmingham

Birmingham, Alabama, United States

RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Valley Children's Hospital

Madera, California, 93636, United States

RECRUITING

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10022, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15219, United States

RECRUITING

Hospital Nacional de Pediatria Garrahan

Buenos Aires, Argentina

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Centre hospitalier universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

ACTIVE NOT RECRUITING

University Hospital Brno

Brno, Czechia

RECRUITING

Rostock University Medical Hospital

Rostock, Germany

RECRUITING

Azienda Ospedaliero-Universitaria Meyer

Florence, Italy

RECRUITING

Prinses Maxima Center

Utrecht, CS, 3584, Netherlands

RECRUITING

Children's Memorial Health Institute

Warsaw, Poland

RECRUITING

Hospital Universitario Cruces

Barakaldo, Spain

RECRUITING

Related Publications (1)

• Weitzman S, Jaffe R. Uncommon histiocytic disorders: the non-Langerhans cell histiocytoses. Pediatr Blood Cancer. 2005 Sep;45(3):256-64. doi: 10.1002/pbc.20246.

  PMID: 15547923 BACKGROUND

MeSH Terms

Conditions

Erdheim-Chester Disease; Histiocytosis, Sinus; Necrobiotic Xanthogranuloma

Condition Hierarchy (Ancestors)

Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Granuloma; Lymphoproliferative Disorders; Necrobiotic Disorders; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Xanthomatosis; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Oussama Abla, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Oussama Abla, MD

CONTACT

416-813-7879; oussama.abla@sickkids.ca

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Target Duration: 10 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Staff Physician

Study Record Dates

• First Submitted: November 3, 2014
• First Posted: November 7, 2014
• Study Start: October 1, 2014
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: June 24, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1); DE (1); US (6); NL (1); AR (1); PL (1); ES (1); CZ (1); CA (2)