- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Journal: Emerging drugs for the treatment of neuromyelitis optica. (Pubmed Central) - Feb 4, 2021
For the first time in this disease, class I treatment evidence is available, but long-term data will be necessary to confirm the overall promising study results of the compounds close to approval. While drug development still centers around AQP4 antibody seropositive patients, current and future research requires consideration of possible diverging treatment demands for the smaller group of seronegative patients and patients with presence of MOG antibodies.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Review, Journal: International consensus guidance for management of myasthenia gravis: 2020 update. (Pubmed Central) - Feb 2, 2021
This study establishes proof of principle that complement inhibition can treat platelet transfusion refractoriness, laying the foundation for a large multicentre trial to assess the overall efficacy of this approach (ClinicalTrials.gov, identifier: NCT02298933). This updated formal consensus guidance of international MG experts, based on new evidence, provides recommendations to clinicians caring for MG patients worldwide.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal, HEOR: Economic Impact of Early-in-Hospital Diagnosis and Initiation of Eculizumab in Atypical Haemolytic Uraemic Syndrome. (Pubmed Central) - Jan 30, 2021
This updated formal consensus guidance of international MG experts, based on new evidence, provides recommendations to clinicians caring for MG patients worldwide. Initiation of eculizumab within 7 days of hospitalisation is associated with lower dialysis rates, less time in ICU, less plasmapheresis, and lower hospitalisation costs compared with late initiation.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: New therapies for neuromyelitis optica spectrum disorder. (Pubmed Central) - Jan 29, 2021
WHERE NEXT?: Despite the rarity of neuromyelitis optica spectrum disorder, a relative abundance of preventive treatment options now exists. In the future, trials should focus on areas of unmet need, including aquaporin-4 seronegative disease, and on development of treatments for acute relapses and for recovery from autoimmune attacks in the CNS.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Second-Generation C5 Inhibitors for Paroxysmal Nocturnal Hemoglobinuria. (Pubmed Central) - Jan 28, 2021
The C5 targeting monoclonal antibody eculizumab has changed the natural history of paroxysmal nocturnal hemoglobinuria (PNH) in the last 10 years...Current strategies encompass increasing drug half-life, developing small molecule inhibitors of C5, and exploring new routes of administration (including subcutaneous and oral agents). In this review, we summarize available data on second-generation C5 inhibitors in PNH, including novel monoclonal antibodies, a small interfering RNA, and small molecules.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Modeling the activation of the alternative complement pathway and its effects on hemolysis in health and disease. (Pubmed Central) - Jan 28, 2021
To demonstrate its clinical applicability, we compared model predictions with clinical observations of the recovery of hematological biomarkers in PNH patients treated with the complement inhibiting anti-C5 antibody eculizumab. In conclusion, the model can enhance the understanding of complement biology and its role in disease pathogenesis, help identifying promising targets for pharmacological intervention, and predict the outcome of complement-targeting pharmacological interventions.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] NMO and antiMOG: Updates on Diagnosis and Treatment (Scientific Program Auditorium) - Jan 27, 2021 - Abstract #ACTRIMSForum2021ACTRIMS_Forum_79;
The availability of proven attack prevention biologics for NMOSD provides new hope for patients with NMOSD. Improved understanding of MOGAD phenotype, natural history and immunopathologic mechanisms of disease combined with optimized MOG-IgG detection assays will allow development of international diagnostic criteria and assist in drug trial design.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, efgartigimod IV (ARGX-113 IV) / argenx, Broteio
Clinical, Review, Journal: Update in immunosuppressive therapy of myasthenia gravis. (Pubmed Central) - Jan 26, 2021
Treatment strategy in MG is quite challenging, mainly due to the disease heterogeneity in terms of clinical presentation, immunopathogenesis and drug response. To solve this dilemma, emerging treatment are based on biological drugs and use new targets of the immune pathway.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Biomarker, Journal: CH50 as a putative biomarker of eculizumab treatment in neuromyelitis optica spectrum disorder. (Pubmed Central) - Jan 26, 2021
Serum C3 and C4 were not dramatically changed during the treatment, in contrast serum CH50 level of each patient had diminished and kept under the detection limit after the treatment without clinical worsening, even in the situation of extending dosing. Serum CH50 level is useful to monitor the drug efficacy during eculizumab treatment.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Evaluation of the initial response in clinical trial efforts for COVID-19 in Brazil. (Pubmed Central) - Jan 21, 2021
The interventions evaluated during the Brazilian research response reflect those of international initiatives, but with a different distribution and a large number of studies assessing hydroxychloroquine/chloroquine. Limitations in methodological design and sample planning represent challenges that could affect the research outreach.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Journal: A reference guide for paroxysmal nocturnal hemoglobinuria: recent updates and points of medical treatment (Pubmed Central) - Jan 16, 2021
With the revised edition of 2020, minor revisions have been added to reflect further advances in treatment and understanding of the disease, while mainly dealing with the clinical introduction of eculizumab derivative, ravulizumab, which uses recycling antibody technology. This review outlines the points of the 2020 revision, including the important points of the previous revision.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Treatment of Rare Inflammatory Kidney Diseases: Drugs Targeting the Terminal Complement Pathway. (Pubmed Central) - Jan 16, 2021
The paradigm of safe and efficacious terminal complement pathway inhibition has been demonstrated by the approval of eculizumab in paroxysmal nocturnal hematuria...This review summarizes the involvement of the terminal effector agents of the complement system in these diseases and provides an overview of inhibitors for complement components C5, C5a, C5aR1, and MAC that are currently in clinical development. Furthermore, a link between increased complement activity and lung damage in severe COVID-19 patients is discussed and the potential for use of complement inhibitors in COVID-19 is presented.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Journal: Acute Rejection Following Kidney Transplantation: State of Art and Future Perspectives. (Pubmed Central) - Jan 13, 2021
Lack of randomized controlled trials and heterogenicity among performed studies limit obtaining definite conclusions. Data about new direct and indirect B cell and plasma cell depleting agents, proximal and terminal complement blockers, IL-6/IL6R pathway inhibitors and antibody removal agents among other promising drugs are reviewed.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Evaluation of the children with C3 glomerulopathy. (Pubmed Central) - Jan 13, 2021
Because of this broad spectrum of disease, treatment may vary widely. We think that complement-targeting therapy with eculizumab should be an alternative option for refractory cases, especially in the early stage of disease, if they did not respond to immunosuppressive treatment.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Review, Journal: B Cells and Antibodies as Targets of Therapeutic Intervention in Neuromyelitis Optica Spectrum Disorders. (Pubmed Central) - Jan 10, 2021
In this article, we review current pathogenetic concepts in NMOSD with a focus on the role of B cells and autoantibodies as major contributors to the propagation of these diseases. Lastly, by highlighting promising experimental and future treatment options, we aim to round up the current state of knowledge on the therapeutic arsenal in NMOSD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, ALPN-101 / AbbVie
[VIRTUAL] Alpn-101 (ICOSL vIgD-Fc), a Dual Antagonist of the ICOS and CD28 Costimulatory Pathways, for Treatment of Steroid Refractory Acute Gvhd (aGVHD): Case Report () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1602;
P1
He received an 11/12 HLA-matched, unrelated peripheral blood stem cell graft after conditioning with fludarabine and melphalan, with neutrophil engraftment by day +28...The post-transplant course was complicated by severe mucositis, difficult oral intake and aspiration leading to PEG tube placement, and biopsy-confirmed lower intestinal tract GVHD diagnosed on day +54 that was refractory to 5 lines of therapy, including high dose methylprednisolone, budesonide and ruxolitinib...The course suggests that GVHD responded briskly to a single dose of ALPN-101 following failure of 5 lines of therapy, despite withdrawal of tacrolimus and an abrupt steroid taper, and prior to the potentially confounding addition of eculizumab. Dual inhibition of CD28 and ICOS, such as by ALPN-101, warrants further clinical study for the treatment and/or prevention of GVHD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, ALPN-101 / AbbVie
[VIRTUAL] Alpn-101 (ICOSL vIgD-Fc), a Dual Antagonist of the ICOS and CD28 Costimulatory Pathways, for Treatment of Steroid Refractory Acute Gvhd (aGVHD): Case Report () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1601;
P1
He received an 11/12 HLA-matched, unrelated peripheral blood stem cell graft after conditioning with fludarabine and melphalan, with neutrophil engraftment by day +28...The post-transplant course was complicated by severe mucositis, difficult oral intake and aspiration leading to PEG tube placement, and biopsy-confirmed lower intestinal tract GVHD diagnosed on day +54 that was refractory to 5 lines of therapy, including high dose methylprednisolone, budesonide and ruxolitinib...The course suggests that GVHD responded briskly to a single dose of ALPN-101 following failure of 5 lines of therapy, despite withdrawal of tacrolimus and an abrupt steroid taper, and prior to the potentially confounding addition of eculizumab. Dual inhibition of CD28 and ICOS, such as by ALPN-101, warrants further clinical study for the treatment and/or prevention of GVHD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, ALPN-101 / AbbVie
[VIRTUAL] Alpn-101 (ICOSL vIgD-Fc), a Dual Antagonist of the ICOS and CD28 Costimulatory Pathways, for Treatment of Steroid Refractory Acute Gvhd (aGVHD): Case Report () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1600;
P1
He received an 11/12 HLA-matched, unrelated peripheral blood stem cell graft after conditioning with fludarabine and melphalan, with neutrophil engraftment by day +28...The post-transplant course was complicated by severe mucositis, difficult oral intake and aspiration leading to PEG tube placement, and biopsy-confirmed lower intestinal tract GVHD diagnosed on day +54 that was refractory to 5 lines of therapy, including high dose methylprednisolone, budesonide and ruxolitinib...The course suggests that GVHD responded briskly to a single dose of ALPN-101 following failure of 5 lines of therapy, despite withdrawal of tacrolimus and an abrupt steroid taper, and prior to the potentially confounding addition of eculizumab. Dual inhibition of CD28 and ICOS, such as by ALPN-101, warrants further clinical study for the treatment and/or prevention of GVHD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, ALPN-101 / AbbVie
[VIRTUAL] Alpn-101 (ICOSL vIgD-Fc), a Dual Antagonist of the ICOS and CD28 Costimulatory Pathways, for Treatment of Steroid Refractory Acute Gvhd (aGVHD): Case Report () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1599;
P1
He received an 11/12 HLA-matched, unrelated peripheral blood stem cell graft after conditioning with fludarabine and melphalan, with neutrophil engraftment by day +28...The post-transplant course was complicated by severe mucositis, difficult oral intake and aspiration leading to PEG tube placement, and biopsy-confirmed lower intestinal tract GVHD diagnosed on day +54 that was refractory to 5 lines of therapy, including high dose methylprednisolone, budesonide and ruxolitinib...The course suggests that GVHD responded briskly to a single dose of ALPN-101 following failure of 5 lines of therapy, despite withdrawal of tacrolimus and an abrupt steroid taper, and prior to the potentially confounding addition of eculizumab. Dual inhibition of CD28 and ICOS, such as by ALPN-101, warrants further clinical study for the treatment and/or prevention of GVHD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, ALPN-101 / AbbVie
[VIRTUAL] Alpn-101 (ICOSL vIgD-Fc), a Dual Antagonist of the ICOS and CD28 Costimulatory Pathways, for Treatment of Steroid Refractory Acute Gvhd (aGVHD): Case Report () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1598;
P1
He received an 11/12 HLA-matched, unrelated peripheral blood stem cell graft after conditioning with fludarabine and melphalan, with neutrophil engraftment by day +28...The post-transplant course was complicated by severe mucositis, difficult oral intake and aspiration leading to PEG tube placement, and biopsy-confirmed lower intestinal tract GVHD diagnosed on day +54 that was refractory to 5 lines of therapy, including high dose methylprednisolone, budesonide and ruxolitinib...The course suggests that GVHD responded briskly to a single dose of ALPN-101 following failure of 5 lines of therapy, despite withdrawal of tacrolimus and an abrupt steroid taper, and prior to the potentially confounding addition of eculizumab. Dual inhibition of CD28 and ICOS, such as by ALPN-101, warrants further clinical study for the treatment and/or prevention of GVHD.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Understanding the Mechanisms of Eculizumab Refractoriness in Transplant Associated Thrombotic Microangiopathy () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1007;
Our data suggest that elevated thrombin generation, especially in patients with intestinal bleeding likely contribute to eculizumab refractoriness in HSCT recipients with TA-TMA. Further studies are required to better understand coagulation and complement pathway interactions in TA-TMA patients in order to determine if eculizumab refractory patients with elevated thrombin levels may benefit from adjunctive complement blocking therapy using MASP-2 inhibitions that potentially can modulate excessive thrombin production (Figure 3).
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Understanding the Mechanisms of Eculizumab Refractoriness in Transplant Associated Thrombotic Microangiopathy () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1006;
Our data suggest that elevated thrombin generation, especially in patients with intestinal bleeding likely contribute to eculizumab refractoriness in HSCT recipients with TA-TMA. Further studies are required to better understand coagulation and complement pathway interactions in TA-TMA patients in order to determine if eculizumab refractory patients with elevated thrombin levels may benefit from adjunctive complement blocking therapy using MASP-2 inhibitions that potentially can modulate excessive thrombin production (Figure 3).
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Understanding the Mechanisms of Eculizumab Refractoriness in Transplant Associated Thrombotic Microangiopathy () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1005;
Our data suggest that elevated thrombin generation, especially in patients with intestinal bleeding likely contribute to eculizumab refractoriness in HSCT recipients with TA-TMA. Further studies are required to better understand coagulation and complement pathway interactions in TA-TMA patients in order to determine if eculizumab refractory patients with elevated thrombin levels may benefit from adjunctive complement blocking therapy using MASP-2 inhibitions that potentially can modulate excessive thrombin production (Figure 3).
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Understanding the Mechanisms of Eculizumab Refractoriness in Transplant Associated Thrombotic Microangiopathy () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1004;
Our data suggest that elevated thrombin generation, especially in patients with intestinal bleeding likely contribute to eculizumab refractoriness in HSCT recipients with TA-TMA. Further studies are required to better understand coagulation and complement pathway interactions in TA-TMA patients in order to determine if eculizumab refractory patients with elevated thrombin levels may benefit from adjunctive complement blocking therapy using MASP-2 inhibitions that potentially can modulate excessive thrombin production (Figure 3).
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Understanding the Mechanisms of Eculizumab Refractoriness in Transplant Associated Thrombotic Microangiopathy () - Jan 9, 2021 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_1003;
Our data suggest that elevated thrombin generation, especially in patients with intestinal bleeding likely contribute to eculizumab refractoriness in HSCT recipients with TA-TMA. Further studies are required to better understand coagulation and complement pathway interactions in TA-TMA patients in order to determine if eculizumab refractory patients with elevated thrombin levels may benefit from adjunctive complement blocking therapy using MASP-2 inhibitions that potentially can modulate excessive thrombin production (Figure 3).
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