INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)

NCT05277272 | Recruiting

• 2 other identifiers: 2021-0821Sobi.HLH-RWE102
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this observational study is to collect data on the natural history of disease of patients with Hemophagocytic Lymphohistiocytosis (HLH) including diagnosis, treatments, responses, and outcomes.

Conditions

Hemophagocytic Lymphohistiocytoses

Outcome Measures

Primary Outcomes (9)

• Time to HLH diagnosis from the initial presentation

  Date of initial presentation and the date of HLH diagnosis as defined by HLH diagnostic criteria (HLH-2004/MAS classification criteria)

  Interval between date of presentation, as defined as the day of appearance of initial HLH symptom, and the date of full HLH diagnosis, as defined by fulfilling the HLH diagnostic criteria, will be measured. Timeframes up to 6 months will be assessed.

• Number of patients with an autoimmune disease at the time of HLH diagnosis

  Presence of an autoimmune disease at the time of diagnosis (e.g., Systemic juvenile idiopathic arthritis, lupus)

  Up to 1 month from HLH diagnosis

• Number of patients with malignancy at the time of HLH diagnosis

  Presence of hematologic and solid malignancies at the time of HLH diagnosis.

  Up to 1 month from HLH diagnosis.

• Number of patients treated with immune-activating agents before HLH diagnosis

  The number of patients treated with immune-activating agents before initial diagnosis (checkpoint inhibitors, CAR-T constructs)

  Up to 1 month before HLH diagnosis.

• Number of patients with central nervous system (CNS) involvement during the HLH disease course.

  CNS involvement as defined by elevated neopterin, white blood cells, or protein at a cerebrospinal fluid or changes in MRI

  Up to 1 month from HLH diagnosis.

• Frequency of a genetic diagnosis underlying the HLH.

  Data on genetic testing will be gathered and investigators will summarize the number to calculate the frequency of a genetic diagnosis.

  Up to 1 month from HLH diagnosis.

• Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis.

  The presence of infections at HLH diagnosis (serology and polymerase chain reaction).

  Up to 1 month from HLH diagnosis.

• Number of patients with organ failure.

  Data will be gathered on organ failure related to HLH (e.g., kidney, lung, CNS).

  Up to 1 year from HLH diagnosis.

• Number of patients with long-term disease-related complications.

  Data on long-term complications (e.g., impaired growth, impaired cognitive development) will be gathered.

  Up to 5 years from HLH diagnosis.

Secondary Outcomes (6)

• Treatment response rate to HLH-related treatments.

  Week two from the start of treatment.

• Time to response to HLH-related therapy for patients in the registry.

  Assessed up to 12 weeks from start of treatment.

• The survival probability of patients in the registry

  From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.

• Number of patients who received hematopoietic stem cell transplantation (HSCT)

  From HLH diagnoses up to 5 years post-HLH diagnosis.

• Frequency of hematopoietic stem cell transplantation (HSCT) related complications

  From HSCT up to 5 years post HSCT.

Study Arms (1)

Patients with clinically suspected or confirmed Hemophagocytic Lymphohistiocytosis

Multi-institutional cohort registry of patients with clinically suspected or confirmed Hemophagocytic Lymphohistiocytosis

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

Patients with clinically suspected or confirmed HLH

You may qualify if:

• Patients with clinically suspected or confirmed HLH, including those meeting the HLH-2004 diagnostic criteria (primary or secondary forms, including malignancy) and other forms of HLH (macrophage activation syndrome \[MAS\], cytokine release syndrome \[CRS\], etc.)
• Signed and dated informed consent and assent (adolescents)

You may not qualify if:

• None

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Sobi, Inc.: collaborator
• Baylor College of Medicine: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Conditions

Lymphohistiocytosis, Hemophagocytic

Condition Hierarchy (Ancestors)

Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Michael Jordan, MD

  Children's Hospital Medical Center, Cincinnati

  STUDY CHAIR

Central Study Contacts

Michael Jordan, MD

CONTACT

(513) 803-9063; Michael.Jordan@cchmc.org

Adi Zoref Lorenz, MD

CONTACT

Adi.Zoref.Lorenz@cchmc.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 29, 2021
• First Posted: March 14, 2022
• Study Start: December 31, 2021
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: February 24, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Beginning three months and ending 5 years following article publication.
• Access Criteria: Proposals should be directed to intohlh@cchmc.org. The requests will be considered by the INTO-HLH steering committee.

Locations

US (1)