| Page 24 | Aplastic Anemia and MDS International Foundation (AAMDSIF)

Granulocyte transfusions in severe aplastic anemia

Author(s):

Rajput RV, Shah V, Shalhoub RN, West-Mitchell K, Cha NR, Conry-Cantilena C, Leitman SF, Young DJ, Wells B, Aue G, Dunbar CE, Patel BA, Childs RW, Young NS, Wu CO, Groarke EM, Kalsi SS

Primary Author:

Rajput RV

Journal Title:

Haematologica

Original Publication Date:

Jun 2024

Patients with severe

Bone Marrow Disease(s):

Aplastic Anemia

FDA Approval of PiaSky for Pediatric Patients with PNH, Featuring Satheesh Chonat, MD

Dr. Satheesh Chonat explains how pediatric patients with PNH, or Paroxysmal Nocturnal Hemoglobinuria, may be treated with PiaSky in this recording. Listen now or read transcript below.

FDA Approval of PiaSky for Patients with PNH

Dr. Carlos DeCastro reviews how PiaSky can be used to treat patients with PNH, or Paroxysmal Nocturnal Hemoglobinuria. Listen or read the transcript below.

Rory Shallis Explains New Therapy for Patients with Low-Risk MDS, Imetelstat

Dr. Rory Shallis explains the treatment, Imetelstat, for patients with Low Risk MDS. Listen or read the transcript below.

NMDP and CIBMTR share new, promising stem cell transplantation trial data using mismatched, unrelated donors at the 2024 EHA Congress

MINNEAPOLIS, June 14, 2024 — NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) announced interim results from the ACCESS trial as an oral abstract during the European Hematology Association (EHA) Annual Meeting in Madrid, Spain.

FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia

On June 6, 2024, the Food and Drug Administration approved imetelstat (Rytelo, Geron Corporation), an oligonucleotide telomerase inhibitor, for adults with low- to intermediate-1 risk

Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials

Author(s):

Peixoto VP, Prudêncio C, Vieira M

Primary Author:

Peixoto VP

Journal Title:

Current Medical Research and Opinion

Original Publication Date:

May 2024

Bone Marrow Disease(s):

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy

Author(s):

Versino F, Fattizzo B

Primary Author:

Versino F, Fattizzo B

Journal Title:

International Journal of Laboratory Hematology

Original Publication Date:

May 2024

Complement inhibitors are the mainstay of

Bone Marrow Disease(s):

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Real-world experience of pegcetacoplan in paroxysmal nocturnal hemoglobinuria

Author(s):

Griffin M, Kelly R, Brindel I, Maafa L, Trikha R, Muus P, Munir T, Varghese AM, Mitchell L, Nagumantry S, Gandhi S, Pike A, Kulasekararaj AG, Peffault de Latour R

Primary Author:

Griffin M

Journal Title:

American Journal of Hematology

Original Publication Date:

May 2021

Pegcetacoplan significantly improves outcomes for patients with

Bone Marrow Disease(s):

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Toward a more patient-centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Author(s):

Efficace F, Buckstein R, Abel GA, Giesinger JM, Fenaux P, Bewersdorf JP, Brunner AM, Bejar R, Borate U, DeZern AE, Greenberg P, Roboz GJ, Savona MR, Sparano F, Boultwood J, Komrokji R, Sallman DA, Xie Z, Sanz G, Carraway HE, Taylor J, Nimer SD,

Primary Author:

Efficace F

Journal Title:

HemaSphere

Original Publication Date:

May 2024

Notable treatment advances have been made in recent years for patients with

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)