What is the efficacy of Anakinra in Hemophagocytic Lymphohistiocytosis (HLH)?

From the Guidelines

Anakinra is not a standard treatment for Hemophagocytic Lymphohistiocytosis (HLH) and its efficacy is uncertain.

Key Considerations

• Due to the lack of strong evidence, it should be used with caution and only in specific cases where other treatments have failed or are not tolerated 1.
• If considered, the typical dose of anakinra is 2 to 6 mg/kg up to 10 mg/kg per day subcutaneously in divided doses, as suggested in the management of MAS-HLH 1.
• Close monitoring of the patient's condition and potential side effects is essential.
• It is recommended to consult with a specialist in HLH management before initiating anakinra therapy.

Additional Context

• Anakinra has been used in other conditions, such as Still's disease, with a reassuring safety profile 1.
• The use of IL-1 inhibitors, such as anakinra, is recommended in Still's disease due to their high evidence of efficacy 1.
• However, the evidence for anakinra in HLH is limited, and its use should be approached with caution, considering the high mortality rate associated with HLH, especially in patients with underlying malignancies 1.

From the Research

Efficacy of Anakinra in Hemophagocytic Lymphohistiocytosis (HLH)

• Anakinra has been shown to be effective in treating HLH, with a response rate of 90.5% in one study 2.
• A retrospective analysis found that anakinra was associated with higher response rates and longer survival compared to etoposide-based therapies 3.
• Another study found that anakinra resulted in a significantly higher 1-year overall survival compared to etoposide-based therapies 4.
• Anakinra has been used as a first-line therapy for HLH, with or without dexamethasone, and has been shown to be well-tolerated and effective in achieving remission 5, 2.

Comparison to Etoposide-Based Therapies

• Anakinra has been compared to etoposide-based therapies in several studies, with results suggesting that anakinra may be a more effective treatment option for HLH 3, 4.
• One study found that anakinra was associated with a higher cumulative incidence of response at 30 days compared to etoposide-based therapies 3.
• Another study found that anakinra resulted in a significantly higher 1-year overall survival compared to etoposide-based therapies 4.

Safety and Tolerability

• Anakinra has been shown to be generally safe and well-tolerated in patients with HLH, with few adverse effects reported 5, 2, 6.
• One study found that anakinra was discontinued due to side effects in only 14.3% of patients 2.