Clinical trial NCT05277272: INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)
Official title: INTO-HLH- Insight Into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (HLH): A Disease Registry for Patients With HLH
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophagocytic Lymphohistiocytoses
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Sobi, Inc.
name: Baylor College of Medicine
First posted: 2022-03-14
Enrollment: 200
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with clinically suspected or confirmed HLH

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.

Detailed description: Hemophagocytic Lymphohistiocytosis (HLH) is a complex, hyperinflammatory syndrome resulting from the interplay of genetic predisposition and various environmental factors. Despite available treatment options for HLH, approximately 30% of patients do not respond to therapy. Moreover, the standard therapy is constrained by its toxicities, and safer treatments are pursued.

There is an unmet need for a deeper understanding of the natural history, clinical/etiologic diversity, complications, and treatment outcomes of patients with HLH, specifically from North America. The proposed study, a collaboration between Cincinnati Children's Hospital Medical Center (CCHMC), Texas Children's Hospital, and Sobi Inc. aims to establish a robust registry that will enable investigators to better define the natural history of HLH.

Eligibility criteria: Inclusion Criteria:

* 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)

Exclusion Criteria:

* None

Primary outcomes:
measure: Time to HLH diagnosis from the initial presentation
timeFrame: 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.
measure: Number of patients with an autoimmune disease at the time of HLH diagnosis
timeFrame: Up to 1 month from HLH diagnosis
measure: Number of patients with malignancy at the time of HLH diagnosis
timeFrame: Up to 1 month from HLH diagnosis.
measure: Number of patients treated with immune-activating agents before HLH diagnosis
timeFrame: Up to 1 month before HLH diagnosis.
measure: Number of patients with central nervous system (CNS) involvement during the HLH disease course.
timeFrame: Up to 1 month from HLH diagnosis.
measure: Frequency of a genetic diagnosis underlying the HLH.
timeFrame: Up to 1 month from HLH diagnosis.
measure: Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis.
timeFrame: Up to 1 month from HLH diagnosis.
measure: Number of patients with organ failure.
timeFrame: Up to 1 year from HLH diagnosis.
measure: Number of patients with long-term disease-related complications.
timeFrame: Up to 5 years from HLH diagnosis.
Secondary outcomes:
measure: Treatment response rate to HLH-related treatments.
timeFrame: Week two from the start of treatment.
measure: Time to response to HLH-related therapy for patients in the registry.
timeFrame: Assessed up to 12 weeks from start of treatment.
measure: The survival probability of patients in the registry
timeFrame: From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.
measure: Number of patients who received hematopoietic stem cell transplantation (HSCT)
timeFrame: From HLH diagnoses up to 5 years post-HLH diagnosis.
measure: Frequency of hematopoietic stem cell transplantation (HSCT) related complications
timeFrame: From HSCT up to 5 years post HSCT.
measure: Number of participants with treatment-related adverse events >/= 3 as assessed by CTCAE 5.0
timeFrame: From initiation of HLH related treatment up to 30 days following discontinuation of treatment.
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Michael Jordan, MD
role: STUDY_CHAIR
References: