- |||||||||| Gamifant (emapalumab-lzsg) / SOBI, Kineret (anakinra) / SOBI
Review, Journal: Does your unwell patient have haemophagocytic lymphohistiocytosis? (Pubmed Central) - Apr 5, 2024 Etoposide-based regimens are sometimes needed, and newer therapies such as emapalumab and JAK inhibitors are increasingly being used...Although relatively rare, haemophagocytic lymphohistiocytosis can be encountered by a broad range of hospital physicians, so knowing how to diagnose and treat this condition is essential. This article reviews the pathogenesis, clinical features, causes, diagnosis and treatment of haemophagocytic lymphohistiocytosis to improve physician recognition and management of this condition to improve future patient outcomes.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI, Kineret (anakinra) / SOBI, MAS825 / Novartis
Macrophage Activation Syndrome (A7) - Mar 17, 2024 - Abstract #EULAR2024EULAR_415; progressive improvement of clinical condition and normalization of laboratory parameters. Glucocorticoids were completely discontinued after five infusions of MAS825.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
A CASE OF PERIPHERAL T-CELL LYMPHOMA PRESENTING AS ACUTE LIVER FAILURE (ePosters - DDW Online Platform) - Mar 14, 2024 - Abstract #DDW2024DDW_1765; He was started on broad spectrum antibiotics and empiric N-Acetylcysteine therapy...The patient's hematologic work-up included a bone marrow biopsy, peripheral blood flow cytometry, and a skin biopsy, which confirmed peripheral T-cell lymphoma (PTCL) and the patient was initiated on chemotherapy (toposide, prednisone, cyclophosphamide, and doxorubin), emapalumab, and steroids for HLH induced by PTCL...The treatment of ALF includes supportive care with vasopressors, renal replacement therapy, and antibiotics if needed, as well as management of the underlying cause. This case highlights a unique and uncommon presentation of peripheral T cell lymphoma associated HLH, in which his only presenting symptom was acute liver failure.
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RATE OF PEDIATRIC RED BLOOD CELL ALLOIMMUNIZATION IS COMPARABLE TO ADULTS (Carpenter Hall) - Dec 13, 2023 - Abstract #WRMC2024WRMC_478; This work provides data to blood banks in the US primarily treating pediatric patients on the type of RBC antibodies to expect and reagents to keep for immunohematology testing. It also provides a foundation for further research into factors associated with alloimmunization in pediatric patients.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Emapalumab to Prevent Graft Failure in High-Risk Patients: The Experience of Stanford Children's Health () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_1313; The successful outcomes observed in 2 patients who received emapalumab prior to their 1st HSCT suggests that administering emapalumab earlier - potentially before the GF process initiates, may yield the greatest benefits. In this high-risk population, a synergistic approach combining emapalumab and eculizumab may hold promise in mitigating the risk of GF.
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Journal: Survival in primary hemophagocytic lymphohistiocytosis 2016-2021: etoposide is better than its reputation. (Pubmed Central) - Nov 22, 2023 Importantly, also in this study, early HSCT of asymptomatic patients resulted in excellent survival (100%), emphasizing the potential benefit of newborn screening. This contemporary standard-of-care study of pHLH patients reveals that first-line etoposide-based therapy is better than previously reported, providing a benchmark for novel treatment regimes.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Journal, Cytokine release syndrome: Schuelke MR, Bassiri H, Behrens EM, et (Pubmed Central) - Nov 15, 2023 This contemporary standard-of-care study of pHLH patients reveals that first-line etoposide-based therapy is better than previously reported, providing a benchmark for novel treatment regimes. No abstract available
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CD22 CAR T Cell-Related IEC-HS Is Associated with an IFN-? Cytokine Signature (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6296; P1 Additional agents for grade 4 IEC-HS included anti-thymoglobulin, dasatanib and emapalumab...These cytokines may be amenable to therapeutic intervention and further study of approaches targeting JAK/STAT and IFN-? are warranted.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Emapalumab for Treatment of Impending Graft Failure (SDCC - Room 11) - Nov 3, 2023 - Abstract #ASH2023ASH_1024; EMA was well tolerated in all patients. Efficacy conclusions are preliminary and limited by the study design, however our treatment study successfully identified a cohort of patients with a markedly high graft failure rate (44%) and we show preliminary evidence to suggest emaplaumab prevented graft failure in patients who had signs of impending graft failure at the time of treatment.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Trial termination: A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH (clinicaltrials.gov) - Oct 6, 2023 P2/3, N=7, Terminated, Efficacy conclusions are preliminary and limited by the study design, however our treatment study successfully identified a cohort of patients with a markedly high graft failure rate (44%) and we show preliminary evidence to suggest emaplaumab prevented graft failure in patients who had signs of impending graft failure at the time of treatment. Active, not recruiting --> Terminated; Sponsor Decision
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Does the H Stand for Herpes Simplex or Hemophagocytic Lymphohistiocytosis? A Challenging Case of Acute Liver Failure (Exhibit Hall) - Jul 29, 2023 - Abstract #ACG2023ACG_3043; The patient was started on IV methylprednisolone for presumed HLH...Within the first day, ferritin levels dropped to 365 though he continued to deteriorate clinically with worsening hemodynamic instability, respiratory failure, and encephalopathy despite addition of etoposide and emapalumab...Despite challenges, it is imperative to distinguish these two entities due to risks of worsening infection with HLH-directed immunosuppression. We recommend a high degree of suspicion for HSV-1 in febrile patients with ALF, even if immunocompetent, as early acyclovir therapy has the potential to improve outcomes.
- |||||||||| Gamifant (emapalumab) / SOBI, Jakafi (ruxolitinib) / Novartis, Incyte
Preclinical, Journal: Cellular and transcriptional impacts of Janus kinase and/or IFN-gamma inhibition in a mouse model of primary hemophagocytic lymphohistiocytosis. (Pubmed Central) - May 29, 2023 Towards this end, ruxolitinib treatment or IFNg neutralization (aIFNg) lessens immunopathology in a model of pHLH in which perforin-deficient mice (Prf1-/-) are infected with Lymphocytic Choriomeningitis virus (LCMV)...When administered at the doses used in this study, the combination of ruxolitinb and aIFNg appears no better than treatment with either drug alone in lessening inflammation. Further studies are warranted to elucidate the optimal doses, schedules, and combinations of these agents for the treatment of patients with pHLH.
- |||||||||| Gamifant (emapalumab) / SOBI
Journal: Efficacy and safety of emapalumab in macrophage activation syndrome. (Pubmed Central) - May 15, 2023 P2, P2/3 with emapalumab was efficacious in inducing remission of MAS secondary to sJIA or AOSD in patients who had failed high-dose glucocorticoids. Screening for viral infections should be performed, particularly for cytomegalovirus.
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Acute Liver Failure Following Lamotrigine Use: Drug Reaction, Hemophagocytic Syndrome, or Both? (Walter E. Washington Convention Center, Area F, Hall C (Lower Level)) - Mar 25, 2023 - Abstract #ATS2023ATS_5232; Nonetheless, there should remain a low threshold to evaluate for HLH in patients with presumed DRESS who are critically ill or have steroid-refractory disease. In these cases, HScore - through appropriately weighted clinical features rather than binary criteria - is a superior scoring tool to HLH-2004 criteria.
- |||||||||| Gamifant (emapalumab) / SOBI
Pediatric CLIPPERS? Think Again! (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_2157; Unfortunately, a rapid tapering of prednisone resulted in disease recurrence...He was confirmed to have FLH, initiated emapalumab therapy, and received a hematopoietic stem cell transplantation...While FHL is more likely in infancy, pediatric CLIPPERS is rare and calls for further investigations to reach a final diagnosis. However, FHL was recently reported in adults thanks to the continuous advancements in genetic testing for HLH, helping clinicians expedite the diagnosis and management of patients with HLH.
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