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Indication

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.

Indication

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.

Important Safety Information

Infections

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.

Increased Risk of Infection With Use of Live Vaccines

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

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.

Adverse Reactions

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.

Click here for full Prescribing Information for Gamifant.

You may also contact Sobi at medinfo.us@sobi.com or 866-773-5274.

References

  1. Gamifant [prescribing information]. Stockholm, Sweden: Swedish Orphan Biovitrum AB.
  2. Marsh RA, Haddad E. How I treat primary haemophagocytic lymphohistiocytosis. Br J Haematol. 2018;182(2):185-199. doi: 10.1111/bjh.15274.
  3. Henter J, Horne A, Aricò M, et al. HLH-2004: Diagnostic and Therapeutic Guidelines for Hemophagocytic Lymphohistiocytosis. Pediatr Blood Cancer. 2007;48:124–131.
  4. Sepulveda F, de Saint Basile G. Hemophagocytic syndrome: primary forms and predisposing conditions. Curr Opin Immunol. 2017; 49:20-26. http://dx.doi.org/10.1016/j.coi.2017.08.004.
  5. Jordan MB, Allen CE, Weitzman S, Filipovich AH, McClain KL. How I treat hemophagocytic lymphohistiocytosis. Blood. 2011;118(15):4041-4052. doi:https://doi.org/10.1182/blood-2011-03-278127.
  6. Morimoto A, Nakazawa Y, Ishii E. Hemophagocytic lymphohistiocytosis: pathogenesis, diagnosis, and management. Pediatr Int. 2016;58(9):817-825.
  7. HLH diagnosis strategy. Cincinnati Children’s Hospital. https://www.cincinnatichildrens.org/service/h/hlh/clinical/test. Accessed June 12, 2021.
  8. Gurunathan A, Boucher A, Mark M, et al. Limitations of HLH-2004 criteria in distinguishing malignancy-associated hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2018;65:e27400. https://doi.org/10.1002/pbc.27400
  9. La Rosée P. Alleviating the storm: ruxolitinib in HLH. Blood. 2016;127(13):1626-1627. doi:10.1182/blood-2016-02-697151.
  10. Lehmberg K, Nichols KE, Henter J-I, et al. Consensus recommendations for the diagnosis and management of hemophagocytic lymphohistiocytosis associated with malignancies. Haematologica. 2015:100(8):997-1004.

Although scientific consensus of the appropriate diagnostic criteria continues to evolve, 3 recognized options for confirming a diagnosis of primary HLH exist1-4:
 

  • The fulfillment of 5 of the 8 HLH-2004 criteria in the absence of an underlying cause, such as malignancy
     
  • A positive genetic test for mutations associated with primary HLH
     
  • Family history consistent with primary HLH
     
Physician holding a baby
  • Fever
  • Hypertriglyceridemia (fasting, ≥265 mg/dL) and/or hypofibrinogenemia (≤1.5 g/L)
  • Splenomegaly
  • Hemophagocytosis* in bone marrow, spleen, or lymph nodes
  • Cytopenias (affecting at least 2 of 3 lineages in the peripheral blood)
    • Hemoglobin <90 g/L (in infants <4 weeks: hemoglobin <100 g/L)
    • Platelets <100 x 109/L
    • Neutrophils <1.0 x 109/L
  • *Note that hemophagocytosis is not specific nor always present in early stages of the disease.
  • Fever
  • Hypertriglyceridemia (fasting, ≥265 mg/dL) and/or hypofibrinogenemia (≤1.5 g/L)
  • Splenomegaly
  • Hemophagocytosis* in bone marrow, spleen, or lymph nodes
  • Cytopenias (affecting at least 2 of 3 lineages in the peripheral blood)
    • Hemoglobin <90 g/L (in infants <4 weeks: hemoglobin <100 g/L)
    • Platelets <100 x 109/L
    • Neutrophils <1.0 x 109/L
  • Ferritin ≥500 μg/L
  • Low or absent natural killer (NK)-cell activity
  • Soluble CD25 (interleukin [IL]-2 receptor) >2400 U/mL (or per local reference laboratory)
*Note that hemophagocytosis is not specific nor always present in early stages of the disease.5
  • FHL3 - UNC13D
  • X-linked lymphoproliferative disorder 1
  • FHL2 - PRF1
  • X-linked lymphoproliferative disorder 2
  • FHL1 - Unknown
  • Griscelli syndrome type 2 (RAB27A)
  • FHL5 - STXBP2 (UNC18B)
  • Chediak-Higashi syndrome - LYST
  • FHL4 - STX11

Accelerate diagnosis with alternatives to genetic testing

When it comes to treating primary HLH, there is no time to wait. Prior to receiving the results of a genetic test, ancillary testing and flow cytometry can be used, where available, to help differentiate between primary and secondary HLH. This approach helps find the triggers or underlying causes of HLH and prevents misdiagnosis.3,5,7,8

Consider running the following tests3:

  • Computed tomography (CT) of chest/abdomen/neck
  • Magnetic resonance imaging (MRI) of brain
  • Viral polymerase chain reactions (PCRs)
    (eg, EBV, CMV, adenovirus)
  • Positron emission tomography (PET)-CT to evaluate lymphoma
  • Test for tick- or mosquito-borne diseases in areas at risk

Consider checking for decreased levels of3,7:

  • Perforin/granzyme B
  • XIAP protein (in males)
  • SAP protein (in males)
  • CD107a

IFNγ is a driver of primary HLH

IFNγ is central to the "cytokine storm"—an uncontrolled release of inflammatory cytokines and overactivation of phagocytes that give the syndrome its name.9

Learn more