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About 1,195 results

ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center

Research & Reviews  394 results

A Case of Paroxysmal Cold Hemoglobinuria Possessing Moderate Paroxysmal Nocturnal Hemog...
https://doi.org/10.12659/AJCR.933102
The American Journal of Case Reports; Sugimoto T, Masui E et. al.

Nov 26th, 2021 - BACKGROUND Paroxysmal cold hemoglobinuria (PCH) is an autoimmune hemolytic disease caused by the Donath-Landsteiner (DL) antibody. Paroxysmal nocturnal hemoglobinuria (PNH) is a non-autoimmune hemolytic disease that is caused by a dysfunction in t...

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
https://clinicaltrials.gov/ct2/show/NCT02946463

Nov 24th, 2021 - The study consisted of a 4-week screening period and a 26-week randomized treatment period (Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, ...

Dysplasia and PNH-type cells in bone marrow aspirates of myelodysplastic syndromes.
https://doi.org/10.1002/cyto.b.22038
Cytometry. Part B, Clinical Cytometry; Westers TM, Alhan C et. al.

Nov 23rd, 2021 - Flow cytometry is increasingly applied in cytopenic patients suspected for myelodysplastic syndromes (MDS). Analysis includes evaluation of antigen expression patterns in granulocytes of which, for example, partial lack of CD16 may indicate dyspla...

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT05131204

Nov 23rd, 2021 - The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or...

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia
https://clinicaltrials.gov/ct2/show/NCT05126849

Nov 19th, 2021 - Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improve...

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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  28 results

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
https://clinicaltrials.gov/ct2/show/NCT02946463

Nov 24th, 2021 - The study consisted of a 4-week screening period and a 26-week randomized treatment period (Primary Evaluation Period). After completion of the 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, ...

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT05131204

Nov 23rd, 2021 - The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or...

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia
https://clinicaltrials.gov/ct2/show/NCT05126849

Nov 19th, 2021 - Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improve...

Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China
https://clinicaltrials.gov/ct2/show/NCT05125341

Nov 18th, 2021 - This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis. This observational study consists of two parts, one part is retrospective study which aims to collect medical ...

Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
https://clinicaltrials.gov/ct2/show/NCT03896152

Oct 28th, 2021 - LNP023 is a novel oral small molecular weight compound, that inhibits alternative complement pathway (AP). Blockade of the AP with oral LNP023 has the potential to prevent both intra - and extravascular hemolysis. This three-period study includes:...

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News  48 results

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...

Phase 3 Drug Trial for ALS Halted for Lack of Efficacy
https://www.medscape.com/viewarticle/957004

Aug 20th, 2021 - A global phase 3 trial of ravulizumab (Ultomiris) for adults with amyotrophic lateral sclerosis (ALS) is being discontinued following a data review, its manufacturer has announced. The CHAMPION-ALS placebo-controlled trial had enrolled 382 adults ...

FDA Expands Approval of Ravulizumab-cwvz in Paroxysmal Nocturnal Hemoglobinuria to Include Pediatric Patients
https://www.onclive.com/view/fda-expands-approval-of-ravulizumab-cwvz-in-paroxysmal-nocturnal-hemoglobinuria

Jun 22nd, 2021 - The FDA has approved the expanded use of ravulizumab-cwvz (Ultomiris) to include children aged 1 month and older, as well as adolescents, with paroxysmal nocturnal hemoglobinuria. The decision was based on interim findings of a phase 3 trial, whi...

FDA Approves New Treatment Option for Rare Anemia
https://www.medscape.com/viewarticle/951476

May 19th, 2021 - A rare, life-threatening anemia now has a new treatment option. The Food and Drug Administration announced the approval of pegcetacoplan (Empaveli) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan is the firs...

VEXAS Syndrome: Implications for Dermatologists
https://www.medscape.com/viewarticle/949785

Apr 22nd, 2021 - When I was a medical student, I always found it gratifying when there was a unifying mechanism that explained the symptoms of a disease. Part of the reason I chose dermatology as a specialty was how frequently we are able to "see" these mechanisms...

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Patient Education  1 results see all →