ArticleHaematologica2021
Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.
Article in Haematologica, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 2 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
16 citing papers in PubMed, 2 syntheses or guidelines pooled it, 19 citations in OpenAlex.
- Real-world use of emapalumab in hemophagocytic lymphohistiocytosis: a scoping review of published evidence.Frontiers in pharmacology · 2026Pooled it
- HLH as an additional warning sign of inborn errors of immunity beyond familial-HLH in children: a systematic review.Frontiers in immunology · 2024Pooled it
- Emapalumab for the Treatment of Immune-Mediated Graft Failure after HSCT.Bone marrow transplantation · 2025Article
- Hemophagocytic lymphohistiocytosis: current treatment advances, emerging targeted therapy and underlying mechanisms.Journal of hematology & oncology · 2024Review
- Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.Nature medicine · 2024Article
- Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.Biomedicines · 2023Article
- Efficacy and safety of emapalumab in macrophage activation syndrome.Annals of the rheumatic diseases · 2023Article
- Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.Frontiers in immunology · 2023Review
- Features of Hemophagocytic Lymphohistiocytosis in Infants With Severe Combined Immunodeficiency: Our Experience From Chandigarh, North India.Frontiers in immunology · 2022Article
- Graft rejection markers in children undergoing hematopoietic cell transplant for bone marrow failure.Blood advances · 2021Article
- Targeting interferon-γ in hyperinflammation: opportunities and challenges.Nature reviews. Rheumatology · 2021Review
- Characteristics and predictors of post-transplant-associated hemophagocytic lymphohistiocytosis in adults.International journal of hematology · 2021Article
- Disseminated Tuberculosis Associated Hemophagocytic Lymphohistiocytosis in a Pregnant Woman With Evans syndrome: A Case Report and Literature Review.Frontiers in immunology · 2021Review
- Possible roads to improve hemophagocytic lymphohistiocytosis outcome.Blood advances · 2020Article
- Identification of New Soluble Factors Correlated With the Development of Graft Failure After Haploidentical Hematopoietic Stem Cell Transplantation.Frontiers in immunology · 2020Article
- Novel Therapeutic Approaches to Familial HLH (Emapalumab in FHL).Frontiers in immunology · 2020Review
Corrections and comments
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Authors and funding
25 authors at 6 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Emapalumab, a fully human anti-IFNγ monoclonal antibody, has been approved in the US as second-line treatment of primary hemophagocytic lymphohistiocytosis (HLH) patients and has shown promise in patients with graft failure (GF) requiring a second allogeneic hematopoietic stem cell transplantation (HSCT). The blockade of IFNγ activity may increase the risk of severe infections, including fatal mycobacteriosis. We report a case of secondary HLH-related GF in the context of HLA-haploidentical HSCT successfully treated with emapalumab in the presence of concomitant life-threatening infections, including disseminated tuberculosis (TB). A 4 years old girl with Adenosine Deaminase-Severe Combined Immunodeficiency complicated by disseminated TB came to our attention for ex-vivo hematopoietic stem cell-gene therapy. After engraftment failure of gene corrected cells, she received two HLA-haploidentical T-cell depleted HSCT from the father, both failed due to GF related to concomitant multiple infections and secondary HLH. Emapalumab administration allowed to control HLH, as well as to prevent GF after a third haplo-HSCT from the mother. Remarkably, all infections improved with antimicrobial medications and disseminated TB did not show any reactivation. This seminal case supports emapalumab use for treatment of secondary HLH and prevention of GF in patients undergoing haplo-HSCT even in the presence of multiple infections, including TB.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.