Analysis of Real-World Data
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Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary... hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.
Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB)... Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.
Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.
During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.
Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.
Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.
Infusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.
In the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).
Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.
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REAL-HLH is the first real-world study to describe the clinical characteristics, treatment patterns, and outcomes of Gamifant treatment across a large and diverse population of patients who have primary hemophagocytic lymphohistiocytosis (HLH).
< VIEW PIVOTAL TRIAL RESULTSA retrospective medical chart review was conducted across 33 US hospitals to identify patients treated with ≥1 dose of Gamifant between Nov 20, 2018, and Oct 31, 2021.
Given the complexity of the disease and risk for misclassification, a consistent rule was applied across all enrolled HLH patients and reviewed by a multispecialty panel of experts. Patients were classified as having primary HLH if they met at least 1 of 3 of the following criteria without evidence of underlying malignancy, rheumatologic, or metabolic disease: at least 5 of 8 HLH-2004 diagnostic criteria OR a family history of HLH OR a known genetic mutation associated with primary HLH.
HSCT=hematopoietic stem cell transplantation.
*The REAL-HLH study was a retrospective, noninterventional medical chart review and was not evaluated by the FDA to grant Gamifant approval. Data were extracted from time of Gamifant initiation to end of data availability, end of study (Dec 31, 2021), or death, whichever occurred first.
46 Patients
were included in the analysis
age range: 0.3-21 years old, median: 1 year
39 CHILDREN
0.3-10 years old
7 ADOLESCENTS AND ADULTS
12-21 years old
90%
(27/30) had ≥5 out of 8 HLH-2004 diagnostic criteria
92% (23/25)
80% (4/5)
54.3%
(25/46) had active infections† at diagnosis
51.3% (20/39)
71.4% (5/7)
21.7%
(10/46) had CNS involvement at diagnosis
23.1% (9/39)
14.3% (1/7)
†Viral infections were the most common (72%, 18/25).
‡The recommended starting dose of Gamifant is 1 mg/kg given as an intravenous infusion over 1 hour twice per week.2 Click here for dosing information.
Limitations of analysis: Due to the study design, information could be missing or incomplete, as data may not be uniformly collected or available across all treatment centers. The constrained availability and inconsistent timing of laboratory assessments further hindered comprehensive evaluation of treatment "response." Additionally, safety data were neither collected nor assessed since the study did not incorporate safety-related endpoints. There may be the possibility of a risk of bias toward patients with poor prognoses, as Gamifant is currently indicated for previously treated patients with primary HLH. As patients were concomitantly administered HLH-related therapies, these findings cannot be solely attributed to Gamifant and may not be generalizable beyond the study cohort. The rarity of primary HLH limited the size of the population in this study and the types of analyses that could be performed, particularly when comparing outcomes between children and adolescents/adults.
(31/42) of patients treated who were eligible for transplantproceeded to HSCT
The top 5 laboratory parameters for which most of the patients achieved normalization were fibrinogen, absolute neutrophil count, platelets, absolute lymphocyte count, and alanine transaminase.
Select the parameter on the graph below that you would like highlighted.§,‖
Fibrinogen
97.4% patients (37/38)
14 days (range: 1-91 days)3
Absolute lymphocyte count
71.4% patients (30/42)
7 days (range: 3-230 days)3
Absolute neutrophil count
88.9% patients (40/45)
7.5 days (range: 3-63 days)3
Alanine transaminase
68.9% patients (31/45)
26 days (range: 2-113 days)3
CXCL9
72.7% patients (24/33)
28.5 days (range: 4-84 days)3
Ferritin
44.4% patients (20/45)
21.5 days (range: 4-112 days)3
Platelets
84.8% patients (39/46)
8 days (range: 3-76 days)3
sCD25
54.1% patients (20/37)
12.5 days (range: 2-84 days)3
§Laboratory parameters for which data were available for ≥50% of the study population.
‖Normalization of laboratory parameters and biomarker values were based on physician report.
The generalizability of reported results may not correlate with results seen in other real-world setting patients. Continuous variables were summarized using mean, standard deviation (SD), median, and interquartile range (IQR). Categorical variables were described using counts and percentages.
Laboratory parameter normalization breakdown
Fibrinogen
97.4% patients (37/38)
14 days (range: 1-91 days)3
Absolute neutrophil count
88.9% patients (40/45)
7.5 days (range: 3-63 days)3
Platelets
84.8% patients (39/46)
8 days (range: 3-76 days)3
Absolute lymphocyte count
71.4% patients (30/42)
7 days (range: 3-230 days)3
Alanine transaminase
68.9% patients (31/45)
26 days (range: 2-113 days)3
CXCL9
72.7% patients (24/33)
28.5 days (range: 4-84 days)3
sCD25
54.1% patients (20/37)
12.5 days (range: 2-84 days)3
Ferritin
44.4% patients (20/45)
21.5 days (range: 4-112 days)3
The results from the REAL-HLH study are consistent with the pivotal trial for Gamifant across age groups.