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Emapalumab’s role in a severe and treatment-resistant paediatric macrophage activation syndrome.

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Title: Emapalumab’s role in a severe and treatment-resistant paediatric macrophage activation syndrome.
Authors: Pereira da Costa, R.1,2 robertopc25@hotmail.com, Lima, M.3, Guedes, S.4, Vieira, M.3, Costa Reis, P.3,5, Claro, A.3, Dias Curado, A.3, Prata, F.3, Esteves, I.3, Campanilho-Marques, R.1,2,5, Marques, J. G.3, Oliveira-Ramos, F.1,2,5
Source: ARP Rheumatology. Jan-Mar2026, Vol. 5 Issue 1, p71-77. 7p.
Subjects: MACROPHAGE activation, DRUG resistance, MONOCLONAL antibodies, CYTOKINE release syndrome, ANTIBODY therapy, DISEASE remission, PEDIATRICS
Abstract: Introduction: Macrophage activation syndrome (MAS) is a life-threatening hyperinflammatory condition. Emapalumab, an IFNg-directed antibody, is approved for use in the USA but not in Europe. Case: A 15-year-old girl presented with fever, odynophagia and a transient rash. After 9 days of hospitalization under empirical antibiotics, she developed pancytopenia, hypofibrinogenaemia, elevated ALT, AST, LDH, triglycerides, soluble CD25, serum calprotectin and ferritin (peak 357,976 ng/ml), and hepatosplenomegaly. Infectious and immune workups were negative, and bone biopsy confirmed haemophagocytosis. MAS was diagnosed, which was complicated by acute respiratory distress and supraventricular tachycardia. High-dose corticosteroids, anakinra and ciclosporin were initiated, with transient improvement. Subsequent drug-induced hepatotoxicity and microangiopathy, and infections worsened her condition. Given refractoriness to standard therapy, emapalumab was started under compassionate use, leading to sustained clinical and laboratory remission. She was discharged and remains stable at six-month follow-up, off corticosteroids and on canakinumab maintenance. Discussion: This case illustrates the challenges of treating severe, refractory MAS. Emapalumab, used for the first time in Portugal, was well tolerated and associated with complete and sustained remission after failure of multiple therapeutic lines. [ABSTRACT FROM AUTHOR]
Copyright of ARP Rheumatology is the property of Sociedade Portuguesa de Reumatologia and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
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  Data: Emapalumab’s role in a severe and treatment-resistant paediatric macrophage activation syndrome.
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  Data: <searchLink fieldCode="AR" term="%22Pereira+da+Costa%2C+R%2E%22">Pereira da Costa, R.</searchLink><relatesTo>1,2</relatesTo><i> robertopc25@hotmail.com</i><br /><searchLink fieldCode="AR" term="%22Lima%2C+M%2E%22">Lima, M.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Guedes%2C+S%2E%22">Guedes, S.</searchLink><relatesTo>4</relatesTo><br /><searchLink fieldCode="AR" term="%22Vieira%2C+M%2E%22">Vieira, M.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Costa+Reis%2C+P%2E%22">Costa Reis, P.</searchLink><relatesTo>3,5</relatesTo><br /><searchLink fieldCode="AR" term="%22Claro%2C+A%2E%22">Claro, A.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Dias+Curado%2C+A%2E%22">Dias Curado, A.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Prata%2C+F%2E%22">Prata, F.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Esteves%2C+I%2E%22">Esteves, I.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Campanilho-Marques%2C+R%2E%22">Campanilho-Marques, R.</searchLink><relatesTo>1,2,5</relatesTo><br /><searchLink fieldCode="AR" term="%22Marques%2C+J%2E+G%2E%22">Marques, J. G.</searchLink><relatesTo>3</relatesTo><br /><searchLink fieldCode="AR" term="%22Oliveira-Ramos%2C+F%2E%22">Oliveira-Ramos, F.</searchLink><relatesTo>1,2,5</relatesTo>
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  Data: <searchLink fieldCode="JN" term="%22ARP+Rheumatology%22">ARP Rheumatology</searchLink>. Jan-Mar2026, Vol. 5 Issue 1, p71-77. 7p.
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  Data: <searchLink fieldCode="DE" term="%22MACROPHAGE+activation%22">MACROPHAGE activation</searchLink><br /><searchLink fieldCode="DE" term="%22DRUG+resistance%22">DRUG resistance</searchLink><br /><searchLink fieldCode="DE" term="%22MONOCLONAL+antibodies%22">MONOCLONAL antibodies</searchLink><br /><searchLink fieldCode="DE" term="%22CYTOKINE+release+syndrome%22">CYTOKINE release syndrome</searchLink><br /><searchLink fieldCode="DE" term="%22ANTIBODY+therapy%22">ANTIBODY therapy</searchLink><br /><searchLink fieldCode="DE" term="%22DISEASE+remission%22">DISEASE remission</searchLink><br /><searchLink fieldCode="DE" term="%22PEDIATRICS%22">PEDIATRICS</searchLink>
– Name: Abstract
  Label: Abstract
  Group: Ab
  Data: Introduction: Macrophage activation syndrome (MAS) is a life-threatening hyperinflammatory condition. Emapalumab, an IFNg-directed antibody, is approved for use in the USA but not in Europe. Case: A 15-year-old girl presented with fever, odynophagia and a transient rash. After 9 days of hospitalization under empirical antibiotics, she developed pancytopenia, hypofibrinogenaemia, elevated ALT, AST, LDH, triglycerides, soluble CD25, serum calprotectin and ferritin (peak 357,976 ng/ml), and hepatosplenomegaly. Infectious and immune workups were negative, and bone biopsy confirmed haemophagocytosis. MAS was diagnosed, which was complicated by acute respiratory distress and supraventricular tachycardia. High-dose corticosteroids, anakinra and ciclosporin were initiated, with transient improvement. Subsequent drug-induced hepatotoxicity and microangiopathy, and infections worsened her condition. Given refractoriness to standard therapy, emapalumab was started under compassionate use, leading to sustained clinical and laboratory remission. She was discharged and remains stable at six-month follow-up, off corticosteroids and on canakinumab maintenance. Discussion: This case illustrates the challenges of treating severe, refractory MAS. Emapalumab, used for the first time in Portugal, was well tolerated and associated with complete and sustained remission after failure of multiple therapeutic lines. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of ARP Rheumatology is the property of Sociedade Portuguesa de Reumatologia and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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        Value: 10.63032/BPDO4893
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        Text: English
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      – SubjectFull: MACROPHAGE activation
        Type: general
      – SubjectFull: DRUG resistance
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      – SubjectFull: MONOCLONAL antibodies
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              Text: Jan-Mar2026
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