Emapalumab as bridge to hematopoietic cell transplant for STAT1 gain-of-function mutations

Kunvarjee, Binni; Bidgoli, Alan; Madan, Rebecca Pellett; Vidal, Esther H.; McAvoy, Devin; Hosszu, Kinga; Scaradavou, Andromachi; Spitzer, Barbara; Curran, Kevin J.; Cancio, Maria; Harris, Andrew C.; O’Reilly, Richard J.; Kung, Andrew L.; Prockop, Susan E.; Boelens, Jaap Jan; Oved, Joseph H.

doi:10.1016/j.jaci.2023.05.016

Cited by 5 publications

(2 citation statements)

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“…142,146,147 It is suggested this effect could be related to increased levels of IFN-γ, which have been implicated in directly inhibiting hematopoietic stem cell function and also inducing Fas expression and increased apoptosis 147 To be clarified which patients should receive it and the timing Emapalumab (monoclonal antibody directed against IFNγ) STAT1 GOF Successfully used in a 6-year-old female with CMC, systemic necrotizing fungemia that caused vocal cord destruction, Mycobacterium abscessus infection, and failure to thrive, affected by a STAT1 GOF mutation (c.629G>A [p.Arg210Lys]) who received an unmodified HSCT from her haploidentical mother. 148 A trial is ongoing to study the effect of emapalumab on inflammation before HSCT and aid in the engraftment process in people with a primary immune regulatory disorder and/or an autoinflammatory condition (NCT05787574)…”

Section: Discussionmentioning

confidence: 99%

Mucocutaneous Candidiasis: Insights Into the Diagnosis and Treatment

Cinicola,

Uva,

Duse

et al. 2024

Pediatric Infectious Disease Journal

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Section: Discussionmentioning

confidence: 99%

Mucocutaneous Candidiasis: Insights Into the Diagnosis and Treatment

Cinicola,

Uva,

Duse

et al. 2024

Pediatric Infectious Disease Journal

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“…IFN-γ played an important role in inducing high rates of graft failure. Emapalumab, a human IFN-γ–blocking monoclonal antibody, has been employed as a pretransplant immunosuppressive strategy in a patient with a STAT1 GOF mutation to facilitate successful engraftment by shielding the graft from inflammation during the process ( 30 ). Furthermore, patients with CARD9 deficiency also achieved complete clinical remission underlying invasive fungal infections after HSCT ( 31 ).…”

Section: Treatmentmentioning

confidence: 99%

Progress in molecular diagnosis and treatment of chronic mucocutaneous candidiasis

Jing,

Liang,

et al. 2024

Front. Immunol.

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Chronic mucocutaneous candidiasis (CMC) is characterized by recurrent or persistent infections with Candida of the skin, nails, and mucous membrane. It is a rare and severe disease resulting from autoimmune defects or immune dysregulations. Nonetheless, the diagnosis and treatment of CMC still pose significant challenges. Erroneous or delayed diagnoses remain prevalent, while the long-term utility of traditional antifungals often elicits adverse reactions and promotes the development of acquired resistance. Furthermore, disease relapse can occur during treatment with traditional antifungals. In this review, we delineate the advancements in molecular diagnostic and therapeutic approaches to CMC. Genetic and biomolecular analyses are increasingly employed as adjuncts to clinical manifestations and fungal examinations for accurate diagnosis. Simultaneously, a range of therapeutic interventions, including Janus kinase (JAK) inhibitors, hematopoietic stem cell transplantation (HSCT), cytokines therapy, novel antifungal agents, and histone deacetylase (HDAC) inhibitors, have been integrated into clinical practice. We aim to explore insights into early confirmation of CMC as well as novel therapeutic options for these patients.

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A semiautomated microfluidic ELISA for the detection of hemophagocytic lymphohistiocytosis biomarkers

Herskovits,

Johnson,

Oved

et al. 2024

American Journal of Clinical Pathology

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Objectives Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by a massive overactivation of the immune system. Because the clinical findings are nonspecific, the development of assays to facilitate rapid diagnosis is critical for patient care. The objectives of this study were to evaluate the performance of a microfluidic enzyme-linked immunosorbent assay (ELISA) for HLH biomarkers and investigate the impact of insourcing this testing on workflow, cost, and turnaround time in a tertiary-care cancer hospital. Methods Trends in order volume were evaluated for C-X-C motif chemokine ligand 9 (CXCL9) and soluble interleukin 2 receptor ɑ (sIL2R), and a microfluidic ELISA was used to measure these analytes in serum samples. Analyte values, turnaround time, and costs were compared for this assay relative to reference laboratory testing. Results Test ordering has increased from 187 to 1030 requests annually over the past 5 years. Insourcing these analytes on a semiautomated ELISA can decrease time to result by approximately 2 days and generate a cost savings of roughly $140,000 annually within our laboratory. Conclusions Using a semiautomated ELISA for sIL2R and CXCL9 may help physicians arrive at a diagnosis and monitor therapy for patients with HLH while decreasing turnaround time and costs within the clinical laboratory.

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Emapalumab as bridge to hematopoietic cell transplant for STAT1 gain-of-function mutations

Cited by 5 publications

References 5 publications

Mucocutaneous Candidiasis: Insights Into the Diagnosis and Treatment

Mucocutaneous Candidiasis: Insights Into the Diagnosis and Treatment

Progress in molecular diagnosis and treatment of chronic mucocutaneous candidiasis

A semiautomated microfluidic ELISA for the detection of hemophagocytic lymphohistiocytosis biomarkers

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