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TOPLINE:
Emapalumab (Gamifant)-containing regimens stabilize key laboratory parameters and show a high 12-month survival probability in patients with rheumatologic disease–associated hemophagocytic lymphohistiocytosis (HLH).
METHODOLOGY:
- Researchers conducted a retrospective medical chart review study across 33 US hospitals to assess the real-world treatment patterns and outcomes in patients with HLH treated with emapalumab.
- They included 15 patients with rheumatologic disease–associated HLH (median age at diagnosis, 5 years; 73.3% women) who received at least one dose of emapalumab between November 20, 2018, and October 31, 2021.
- Most patients with rheumatologic disease–associated HLH had either systemic juvenile idiopathic arthritis (n = 9) or adult-onset Still’s disease (n = 1).
- Patients received emapalumab for refractory, recurrent, or progressive disease, with an overall treatment duration of 63 days.
- The primary objective of this study was to describe emapalumab treatment patterns such as time to initiation, treatment duration, dosing patterns, and reasons for initiation.
TAKEAWAY:
- Most patients (60%) with rheumatologic disease–associated HLH were critically ill and were initiated on emapalumab in an intensive care unit; emapalumab was mostly initiated for treating refractory (33.3%) and recurrent (33.3%) disease.
- All patients concurrently received emapalumab with other HLH-related therapies, with glucocorticoids (100%) and anakinra (60%) used most frequently.
- Emapalumab treatment led to achievement of normal fibrinogen levels (> 360 mg/dL), according to defined laboratory criteria in all patients with rheumatologic disease–associated HLH, and an 80.6% reduction in the required glucocorticoid dose.
- The 12-month survival probability from the initiation of emapalumab was 86.7% in all patients with rheumatologic disease–associated HLH and 90.0% in the subset with systemic juvenile idiopathic arthritis or adult-onset Still’s disease.
IN PRACTICE:
“In this study, emapalumab-containing regimens normalized rheumatologic disease–associated laboratory parameters, substantially reduced glucocorticoid dose, and were associated with low mortality,” the authors wrote.
SOURCE:
The study was led by Shanmuganathan Chandrakasan, MD, Children’s Healthcare of Atlanta, Emory University, Atlanta, Georgia, and was published online on September 8, 2024, in Arthritis & Rheumatology.
LIMITATIONS:
Chart data required for analyses were missing or incomplete in this retrospective study. The sample size of patients with rheumatologic disease–associated HLH was small. No safety data were collected.
DISCLOSURES:
The study was supported by Sobi, which markets emapalumab. Some authors declared receiving grants, consulting fees, or payments or having financial and nonfinancial interests and other ties with several pharmaceutical companies, including Sobi.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
Emapalumab (Gamifant)-containing regimens stabilize key laboratory parameters and show a high 12-month survival probability in patients with rheumatologic disease–associated hemophagocytic lymphohistiocytosis (HLH).
METHODOLOGY:
- Researchers conducted a retrospective medical chart review study across 33 US hospitals to assess the real-world treatment patterns and outcomes in patients with HLH treated with emapalumab.
- They included 15 patients with rheumatologic disease–associated HLH (median age at diagnosis, 5 years; 73.3% women) who received at least one dose of emapalumab between November 20, 2018, and October 31, 2021.
- Most patients with rheumatologic disease–associated HLH had either systemic juvenile idiopathic arthritis (n = 9) or adult-onset Still’s disease (n = 1).
- Patients received emapalumab for refractory, recurrent, or progressive disease, with an overall treatment duration of 63 days.
- The primary objective of this study was to describe emapalumab treatment patterns such as time to initiation, treatment duration, dosing patterns, and reasons for initiation.
TAKEAWAY:
- Most patients (60%) with rheumatologic disease–associated HLH were critically ill and were initiated on emapalumab in an intensive care unit; emapalumab was mostly initiated for treating refractory (33.3%) and recurrent (33.3%) disease.
- All patients concurrently received emapalumab with other HLH-related therapies, with glucocorticoids (100%) and anakinra (60%) used most frequently.
- Emapalumab treatment led to achievement of normal fibrinogen levels (> 360 mg/dL), according to defined laboratory criteria in all patients with rheumatologic disease–associated HLH, and an 80.6% reduction in the required glucocorticoid dose.
- The 12-month survival probability from the initiation of emapalumab was 86.7% in all patients with rheumatologic disease–associated HLH and 90.0% in the subset with systemic juvenile idiopathic arthritis or adult-onset Still’s disease.
IN PRACTICE:
“In this study, emapalumab-containing regimens normalized rheumatologic disease–associated laboratory parameters, substantially reduced glucocorticoid dose, and were associated with low mortality,” the authors wrote.
SOURCE:
The study was led by Shanmuganathan Chandrakasan, MD, Children’s Healthcare of Atlanta, Emory University, Atlanta, Georgia, and was published online on September 8, 2024, in Arthritis & Rheumatology.
LIMITATIONS:
Chart data required for analyses were missing or incomplete in this retrospective study. The sample size of patients with rheumatologic disease–associated HLH was small. No safety data were collected.
DISCLOSURES:
The study was supported by Sobi, which markets emapalumab. Some authors declared receiving grants, consulting fees, or payments or having financial and nonfinancial interests and other ties with several pharmaceutical companies, including Sobi.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
TOPLINE:
Emapalumab (Gamifant)-containing regimens stabilize key laboratory parameters and show a high 12-month survival probability in patients with rheumatologic disease–associated hemophagocytic lymphohistiocytosis (HLH).
METHODOLOGY:
- Researchers conducted a retrospective medical chart review study across 33 US hospitals to assess the real-world treatment patterns and outcomes in patients with HLH treated with emapalumab.
- They included 15 patients with rheumatologic disease–associated HLH (median age at diagnosis, 5 years; 73.3% women) who received at least one dose of emapalumab between November 20, 2018, and October 31, 2021.
- Most patients with rheumatologic disease–associated HLH had either systemic juvenile idiopathic arthritis (n = 9) or adult-onset Still’s disease (n = 1).
- Patients received emapalumab for refractory, recurrent, or progressive disease, with an overall treatment duration of 63 days.
- The primary objective of this study was to describe emapalumab treatment patterns such as time to initiation, treatment duration, dosing patterns, and reasons for initiation.
TAKEAWAY:
- Most patients (60%) with rheumatologic disease–associated HLH were critically ill and were initiated on emapalumab in an intensive care unit; emapalumab was mostly initiated for treating refractory (33.3%) and recurrent (33.3%) disease.
- All patients concurrently received emapalumab with other HLH-related therapies, with glucocorticoids (100%) and anakinra (60%) used most frequently.
- Emapalumab treatment led to achievement of normal fibrinogen levels (> 360 mg/dL), according to defined laboratory criteria in all patients with rheumatologic disease–associated HLH, and an 80.6% reduction in the required glucocorticoid dose.
- The 12-month survival probability from the initiation of emapalumab was 86.7% in all patients with rheumatologic disease–associated HLH and 90.0% in the subset with systemic juvenile idiopathic arthritis or adult-onset Still’s disease.
IN PRACTICE:
“In this study, emapalumab-containing regimens normalized rheumatologic disease–associated laboratory parameters, substantially reduced glucocorticoid dose, and were associated with low mortality,” the authors wrote.
SOURCE:
The study was led by Shanmuganathan Chandrakasan, MD, Children’s Healthcare of Atlanta, Emory University, Atlanta, Georgia, and was published online on September 8, 2024, in Arthritis & Rheumatology.
LIMITATIONS:
Chart data required for analyses were missing or incomplete in this retrospective study. The sample size of patients with rheumatologic disease–associated HLH was small. No safety data were collected.
DISCLOSURES:
The study was supported by Sobi, which markets emapalumab. Some authors declared receiving grants, consulting fees, or payments or having financial and nonfinancial interests and other ties with several pharmaceutical companies, including Sobi.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.