Ravulizumab: A Review in Atypical Haemolytic Uraemic Syndrome

Syed, Yahiya Y.

doi:10.1007/s40265-021-01481-6

Cited by 26 publications

(20 citation statements)

References 29 publications

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“…Liver transplant may be a curative therapy, but has been associated with high mortality risk. Ravulizumab, a longer acting version of eculizumab, has recently been approved for use in aHUS in several jurisdictions (Syed, 2021).…”

Section: Current Management/treatmentmentioning

confidence: 99%

Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence‐Based Approach from the Writing Committee of the American Society for Apheresis: The Ninth Special Issue

Connelly‐Smith

Alquist

Aqui

et al. 2023

J of Clinical Apheresis

223

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The American Society for Apheresis (ASFA) Journal of Clinical Apheresis (JCA) Special Issue Writing Committee is charged with reviewing, updating, and categorizing indications for the evidence-based use of therapeutic apheresis (TA) in human disease. In the Ninth Edition, the JCA Special Issue Writing Committee has incorporated systematic review and evidence-based approaches in the grading of evidence and categorization of apheresis indications to make recommendations on the use of apheresis in a wide variety of diseases and conditions. This edition has largely maintained the general layout and concept of a fact sheet introduced in the Fourth Edition (2007). Each fact sheet succinctly summarizes the evidence for the use of TA in a specific disease or medical condition. The Ninth Edition of the JCA Special Issue comprises 91 fact sheets and

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Section: Current Management/treatmentmentioning

confidence: 99%

Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence‐Based Approach from the Writing Committee of the American Society for Apheresis: The Ninth Special Issue

Connelly‐Smith

Alquist

Aqui

et al. 2023

J of Clinical Apheresis

223

View full text Add to dashboard Cite

show abstract

“…The latter may or may not have a detectable underlying genetic defect in complement regulation, and the C5 inhibitor eculizumab is now approved for aHUS with or without a defined genetic mutation, and it is considered first line therapy (12)(13)(14). Additionally, ravulizumab is a humanized monoclonal antibody derived from eculizumab but with a 3X greater half-life requiring dosing every 4 -8 weeks that is now approved for aHUS (15). There are no trials comparing these 2 C5 inhibitors.…”

Section: Introductionmentioning

confidence: 99%

Thrombotic Microangiopathy, an Unusual Form of Monoclonal Gammopathy of Renal Significance: Report of 3 Cases and Literature Review

Filippone

Newman

et al. 2021

Front. Immunol.

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Monoclonal gammopathies result from neoplastic clones of the B-cell lineage and may cause kidney disease by various mechanisms. When the underlying clone does not meet criteria for a malignancy requiring treatment, the paraprotein is called a monoclonal gammopathy of renal significance (MGRS). One rarely reported kidney lesion associated with benign paraproteins is thrombotic microangiopathy (TMA), provisionally considered as a combination signifying MGRS. Such cases may lack systemic features of TMA, such as a microangiopathic hemolytic anemia, and the disease may be kidney limited. There is no direct deposition of the paraprotein in the kidney, and the presumed mechanism is disordered complement regulation. We report three cases of kidney limited TMA associated with benign paraproteins that had no other detectable cause for the TMA, representing cases of MGRS. Two of the cases are receiving clone directed therapy, and none are receiving eculizumab. We discuss in detail the pathophysiological basis for this possible association. Our approach to therapy involves first ruling out other causes of TMA as well as an underlying B-cell malignancy that would necessitate direct treatment. Otherwise, clone directed therapy should be considered. If refractory to such therapy or the disease is severe and multisystemic, C5 inhibition (eculizumab or ravulizumab) may be indicated as well.

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“…The NCT03518203 trial tested eculizumab in pediatric and adult patients with high risk of developing TA-TMA, but results are not yet known. Ravulizumab is a humanized monoclonal antibody that inhibits complement protein C5, with the same indication as eculizumab for HUS but with a longer half-life [ 75 ]. Two trials are testing Ravalizumab in patients with TA-TMA including children (NCT04557735, NCT04543591).…”

Section: Resultsmentioning

confidence: 99%

The Development of New Agents for Post-Hematopoietic Stem Cell Transplantation Non-Infectious Complications in Children

Ilan

Brivio

Algeri

et al. 2023

JCM

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Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT. These strategies have been mainly carried out in adults and some are now being translated into children. In this manuscript, we review the recent advancements in the development and implementation of treatment options for post-HSCT non-infectious complications in pediatric patients with leukemia and other non-malignant conditions, with a special attention on the new agents available within clinical trials. We focused on the following conditions: graft failure, prevention of relapse and early interventions after detection of minimal residual disease positivity following HSCT in acute lymphoblastic and myeloid leukemia, chronic graft versus host disease, non-infectious pulmonary complications, and complications of endothelial origin.

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Ravulizumab: A Review in Atypical Haemolytic Uraemic Syndrome

Cited by 26 publications

References 29 publications

Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence‐Based Approach from the Writing Committee of the American Society for Apheresis: The Ninth Special Issue

Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence‐Based Approach from the Writing Committee of the American Society for Apheresis: The Ninth Special Issue

Thrombotic Microangiopathy, an Unusual Form of Monoclonal Gammopathy of Renal Significance: Report of 3 Cases and Literature Review

The Development of New Agents for Post-Hematopoietic Stem Cell Transplantation Non-Infectious Complications in Children

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