Blog
The INTO-HLH Registry and its Progress
This week, we are pleased to have team from the INTO-HLH Registry as guests, to speak about the registry, its purpose, and its progress.
The INTO-HLH Registry
The Insight into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (INTO-HLH) registry is a collaborative effort with Cincinnati Children’s Hospital Medical Center (CCHMC), Texas Children’s Hospital, and Sobi North America as the industry sponsor.
What is HLH?
Hemophagocytic Lymphohistiocytosis (HLH) is a hyperinflammatory condition influenced by both genetic factors and environmental triggers. Hyperinflammation means that the immune system is turned on far more intensely than it ever should be, causing life-threatening organ damage. This severe hyperinflammatory syndrome poses significant challenges for diagnosis and treatment.
The INTO-HLH Registry
Over the past 25 years, scientific knowledge and treatment options have made significant strides, including the development of new targeted therapies. Despite these advancements, about 30% of patients still do not respond to existing treatments, highlighting the need for further research. To address this, we established a robust registry to better understand the natural history, treatment patterns, and outcomes of HLH.
This registry aims to provide a deeper understanding of the disease’s natural progression, its diverse causes and complications, and the outcomes of different treatment approaches. With this research, we hope to gain valuable insight into key issues facing patients with HLH, such as:
- Wrong diagnosis
- Delayed or missed diagnosis
- Factors contributing to early mortality (and strategies for intervention)
- Late deaths on therapy (prior to Bone Marrow Transplant)
- Adverse event consequences
- Central Nervous System (CNS) involvement
We aim to enhance our knowledge and improve the care and treatment of those affected by HLH.
Progress Thus Far
Current Enrollment Status
The registry aims to register as many HLH patients as possible to enhance treatment outcomes. We seek to enroll patients with all forms of HLH, including primary HLH, secondary HLH (including malignancy-related), macrophage activation syndrome (MAS), and cytokine release syndrome (CRS). Currently, the registry has over 90 patients consented to participate, with data abstracted from 60 patients. The current registry is heavily weighted toward primary HLH, as shown in Figure 1, and we seek to inform more secondary patients about the existence of the registry and the value they can greatly add to the HLH community by joining.
Our outreach by advocacy partners and patients currently registered to provide awareness of the registry is critical to our advancement. The registry website includes a Patients & Families page where patients can connect with these amazing organizations as part of our Community of Hope.
Initial Data Analysis
The registry will allow us to gather comprehensive data to improve diagnosis and treatment for this population. By describing this detailed data and uncovering correlations, we aim to drive research forward to eventually improve patient outcomes. Significant advancements have been made in identifying HLH’s clinical patterns, which could possibly shorten the time to diagnosis. Ongoing research and data collection are critical.
Future Hopes
The registry hopes to uncover new insights into HLH, leading to more effective treatments. It aims to transform patient care and outcomes by fostering a community of involvement and sharing knowledge. The ultimate hope of this registry is to transition from the opinion and wisdom of experienced physicians to actual data to support the best diagnosis and treatment for this population. This data will enable us to discover correlations across many laboratory values determined at diagnosis and follow the progression through treatment.
Participation:
Patient participation requires consent to understanding how your data will be used and signing a medical record release form to allow sharing of data to the registry. HLH-related data is abstracted from initial disease presentation to diagnosis, through treatment until Hematopoietic Stem Cell Transplantation (HSCT) or the end of HLH-directed treatments. Patients are followed for at least five years to assess short- and long-term outcomes. We invite all patients diagnosed with suspected or confirmed HLH to participate, as well as registration of loved ones lost to HLH by their family members.
Please Join the Fight:
You or your loved one can enroll by visiting the registry website (https://hlhregistry.org/join-the-fight). There, you can learn more about the registry, ask questions, or stay informed by signing up for the HLH Registry Newsletter. Participants can self-enroll through our secure consent portal or, if preferred, contact the INTO-HLH Registry Team (IHRT) by leaving your contact information on the registration form, sending an email to intohlh@cchmc.org, or calling 513-916-7099.
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Our thanks again to the INTO-HLH Registry Team. Please help support their research and share it with friends and family to help get more HLH voices informing future research.
