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About 1,905 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  572 results

Long-Term Outcomes of Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Ec...
https://doi.org/10.1111/ejh.13970
European Journal of Haematology; Versmold K, Alashkar F et. al.

Mar 28th, 2023 - Describe the real-world clinical profile of eculizumab-treated patients by characterizing their short- and long-term clinical and laboratory outcomes. This retrospective study used preexisting medical records of eculizumab-treated patients with pa...

A bispecific inhibitor of complement and coagulation blocks activation in complementopa...
https://doi.org/10.1182/blood.2022019359
Blood Andersen JF, Lei H et. al.

Mar 23rd, 2023 - Inhibitors of complement and coagulation are present in the saliva of a variety of blood feeding arthropods that transmit parasitic and viral pathogens. Here we describe the structure and mechanism of action of the sand fly salivary protein lufaxi...

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused W...
https://clinicaltrials.gov/ct2/show/NCT01174108

Mar 17th, 2023 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

Aplastic Anemia Treatment & Management
http://emedicine.medscape.com/article/198759-treatment

Mar 15th, 2023 - Approach Considerations Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy, or hematopoietic cell transplantation (HCT). Severe and very severe aplastic anemia (SAA and VSAA, respectively; see Workup/Staging...

Bone Marrow Donor Procedure
https://emedicine.medscape.com/article/1948293-overview

Mar 13th, 2023 - Background The history of stem cell transplant goes back as early as 1939, when the first documented clinical transplant was performed. In 1968, the first successful allogenic stem cell transplant was made possible followed by series of achievemen...

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Guidelines  1 results

Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and ...
https://doi.org/10.1002/cyto.b.20525
Cytometry. Part B, Clinical Cytometry; Borowitz MJ, Craig FE et. al.

Jun 10th, 2010 - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem cell disorder characterized by a somatic mutation in the PIGA gene, leading to a deficiency of proteins linked to the cell membrane via glycophosphatidylinositol (GPI) anchors....

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Drugs  6 results see all →

Clinicaltrials.gov  88 results

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused W...
https://clinicaltrials.gov/ct2/show/NCT01174108

Mar 17th, 2023 - Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS)...

Ravulizumab-Controlled Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are C...
https://clinicaltrials.gov/ct2/show/NCT05133531

Mar 3rd, 2023 - The primary objective of the study is: To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 26-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with activ...

Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT03157635

Mar 2nd, 2023 - This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be evaluated in healthy...

A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT05744921

Feb 27th, 2023 - The primary objective of the study is to describe the long-term safety, tolerability, and efficacy of pozelimab and cemdisiran combination therapy in patients with PNH The secondary objectives of the study are to describe the long-term effect of t...

A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibi...
https://clinicaltrials.gov/ct2/show/NCT04432584

Feb 24th, 2023 - A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.

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News  92 results

Azacitidine-Based Combinations Represent New Treatment Possibilities in MDS
https://www.onclive.com/view/azacitidine-based-combinations-represent-new-treatment-possibilities-in-mds

Nov 2nd, 2022 - The exploration of combination therapies with an azacitidine (Vidaza) backbone could alter the treatment landscape for patients with myelodysplastic syndromes (MDS), according Mikkael A. Sekeres, MD, MS. Data are expected soon from the phase 3...

Practical Considerations for Fellows Treating VTE
https://www.onclive.com/view/practical-considerations-for-fellows-treating-vte

Jul 12th, 2022 - Venous thromboembolism (VTE) is a common diagnosis indicating a blood clot located in a vein.1 Most hematology/oncology fellows will care for patients with this diagnosis throughout their training and long after their graduation from fellowship. D...

Pegcetacoplan Improves Efficacy in Complement Inhibitor–Naïve Paroxysmal Nocturnal Hemoglobinuria
https://www.onclive.com/view/pegcetacoplan-improves-efficacy-in-complement-inhibitor-na-ve-paroxysmal-nocturnal-hemoglobinuria

Apr 9th, 2022 - Pegcetacoplan (Empaveli) treatment was associated with hemoglobin stabilization and normalization, as well as a superior change from baseline in hemoglobin levels, which showed a meaningful correction of anemia, in patients with complement inhibit...

Expert Highlights Advances for Patients With PNH
https://www.onclive.com/view/expert-highlights-advances-for-patients-with-pnh

Oct 6th, 2021 - According to Mohamed Kharfan-Dabaja, MD, there is ever-increasing hope for patients suffering from paroxysmal nocturnal hemoglobinuria (PNH), a rare disease characterized by the premature breakdown of blood cells that is caused by a somatic mutati...

European Commission Expands Ravulizumab-cwvz Approval in Paroxysmal Nocturnal Hemoglobinuria With Pediatric Indication
https://www.onclive.com/view/european-commission-expands-ravulizumab-cwvz-approval-in-paroxysmal-nocturnal-hemoglobinuria-with-pediatric-indication

Sep 3rd, 2021 - The European Commission has granted an expanded approval to ravulizumab-cwvz (Ultomiris) to include children with a body weight of at least 10 kg, as well as adolescents, with paroxysmal nocturnal hemoglobinuria (PNH).1 The approval is based on...

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Patient Education  3 results see all →