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Severe breakthrough hemolysis during compassionate use of Pegcetacoplan in paroxysmal nocturnal hemoglobinuria: managing an emergency

Author(s): 
Metafuni E, Carbone A, Petriccione L, Giammarco S, Limongiello MA, Bellesi S, Chiusolo P, Sica S
Primary Author: 
Metafuni E
Journal Title: 
Blood Transfusion
Original Publication Date: 
Jul 2024
Bone Marrow Disease(s): 

Safe, Successful Pregnancies Possible After AlloHCT

Findings refute former consensus that pregnancies post-transplant are nearly impossible, highlight need for increased fertility counseling

 

(WASHINGTON, July 15, 2024) — Despite treatment-related fertility challenges, female patients can become pregnant and give birth to healthy children after undergoing allogeneic hematopoietic cell transplantation (alloHCT), according to a study published in Blood.

Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors

Author(s): 
Panse J, Daguindau N, Okuyama S, Peffault de Latour R, Schafhausen P, Straetmans N, Al-Adhami M, Persson E, Wong RSM
Primary Author: 
Panse J
Journal Title: 
PLOS One
Original Publication Date: 
Jul 2024

Background: Although complement component 5 inhibitors (C5is) eculizumab and ravulizumab improve

Bone Marrow Disease(s): 

Development of a target concentration intervention to individualize paroxysmal nocturnal hemoglobinuria treatment with pegcetacoplan

Author(s): 
Ter Avest M, Langemeijer SMC, Blijlevens NMA, van de Kar NCAJ, Ter Heine R
Primary Author: 
Ter Avest M
Journal Title: 
Annals of Hematology
Original Publication Date: 
Jul 2024

Pegcetacoplan (Aspaveli®/Empaveli™) is a factor C3 inhibitor that is approved for the treatment of

Bone Marrow Disease(s): 

Paroxysmal nocturnal hemoglobinuria-related thrombosis in the era of novel therapies: a 2043-patient-year analysis

Author(s): 
Gurnari C, Awada H, Pagliuca S, Dima D, Ullah F, Kawashima N, Kubota Y, Colak C, Visconte V, Patel BJ, Dhillon V, Marneni N, Balasubramanian SK, Kishtagari A, Bat T, Maciejewski JP
Primary Author: 
Gurnari C
Journal Title: 
Blood
Original Publication Date: 
Jul 2024

Thrombophilia is one of the principal features of

Bone Marrow Disease(s): 

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Author(s): 
Montoro J, Eikema DJ, Tuffnell J, Potter V, Kalwak K, Halkes CJM, Kulagin A, Collin M, Wynn RF, Robinson S, Nicholson E, Sengeloev H, Clay J, Halahleh K, Skorobogatova E, Sanz J, Passweg J, Mielke S, Ryhänen S, Carpenter B, Gedde-Dahl T, Tholouli E,
Primary Author: 
Montoro J
Journal Title: 
Blood
Original Publication Date: 
Jul 2024

Selecting the most suitable alternative donor becomes challenging in severe

Bone Marrow Disease(s): 

Phase 3 randomized COMMODORE 2 trial: Crovalimab versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria naive to complement inhibition

Author(s): 
Röth A, He G, Tong H, Lin Z, Wang X, Chai-Adisaksopha C, Lee JH, Brodsky A, Hantaweepant C, Dumagay TE, Demichelis-Gómez R, Rojnuckarin P, Sun J, Höglund M, Jang JH, Gaya A, Silva F, Obara N, Kelly RJ, Beveridge L, Buatois S, Chebon S, Gentile B,
Primary Author: 
Röth A
Journal Title: 
American Journal of Hematology
Original Publication Date: 
Jun 2024

Crovalimab is a novel C5 complement inhibitor that enables rapid and sustained C5 inhibition with subcutaneous, low-volume self-administration every 4 weeks.

Bone Marrow Disease(s):