| Page 5 | Aplastic Anemia and MDS International Foundation (AAMDSIF)

FDA approval of Tibsovo with Dr. Amir Fathi

Here is a discussion about recent FDA approval of Tibsovo for Relapsed/Refractory MDS with Dr. Amir Fathi, who is an Associate Professor of Medicine at Harvard Medical School and the Director of the Leukemia Program at Massachusetts General Hospital.

Expanded Access to Reblozyl® for Patients with Dr. Guillermo Garcia Manero

This episode focuses on the US Food and Drug Administration expanded access approval of luspatercept or Reblozyl with Dr. Guillermo Garcia-Manero, Chief of the Section of MDS in the Department of Leukemia at MD Anderson Cancer Center in Houston.

Discordant pathologic diagnoses of myelodysplastic neoplasms and their implications for registries and therapies

Author(s):

Gorak EJ, Otterstatter M, Al Baghdadi T, Gillis N, Foran JM, Liu JJ, Bejar R, Gore SD, Kroft SH, Harrington A, Saber W, Starczynowski D, Rollison DE, Zhang L, Moscinski L, Wilson S, Thompson J, Borchert C, Sherman S, Hebert D, Walker ME, Padron E,

Primary Author:

Gorak EJ

Journal Title:

Blood Advances

Original Publication Date:

Oct 2023

Myelodysplastic neoplasms (MDS) are a collection of hematopoietic disorders with widely variable prognoses and treatment options. Accurate pathologic diagnoses present challenges because of interobserver variability in interpreting morphology and quantifying dysplasia.

Bone Marrow Disease(s):

Patterns of Diagnostic Evaluation and Determinants of Treatment in Older Patients With Non-transfusion Dependent Myelodysplastic Syndromes

Author(s):

Mukherjee S, Dong W, Schiltz NK, Stange KC, Cullen J, Gerds AT, Carraway HE, Singh A, Advani AS, Sekeres MA, Koroukian SM

Primary Author:

Mukherjee S

Journal Title:

Oncologist

Original Publication Date:

Oct 2023

Background: Older patients with

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)

Germline predisposition traits in allogeneic hematopoietic stem-cell transplantation for myelodysplastic syndromes: a survey-based study and position paper on behalf of the Chronic Malignancies Working Party of the EBMT

Author(s):

Gurnari C, Robin M, Godley LA, Drozd-Sokołowska J, Włodarski MW, Raj K, Onida F, Worel N, Ciceri F, Carbacioglu S, Kenyon M, Aljurf M, Bonfim C, Makishima H, Niemeyer C, Fenaux P, Zebisch A, Hamad N, Chalandon Y, Hellström-Lindberg E, Voso MT, Mecucci C,

Primary Author:

Gurnari C,

Journal Title:

Lancet Hematology

Original Publication Date:

Oct 2023

The recent application of whole exome or whole genome sequencing unveiled a plethora of germline variants predisposing to myeloid disorders, particularly myelodysplastic neoplasms. The presence of such variants in patients with

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)

Clinical decision‐making and treatment of myelodysplastic syndromes

Author(s):

Hellstrom-Lindberg ES, Kröger N

Primary Author:

Hellstrom-Lindberg ES

Journal Title:

Blood

Original Publication Date:

Oct 2023

The

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)

Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS)

Author(s):

Oliva EN, Riva M, Niscola P, Santini V, Breccia M, Giai V, Poloni A, Patriarca A, Crisà E, Capodanno I, Salutari P, Reda G, Cascavilla N, Ferrero D, Guarini A, Tripepi G, Iannì G, Russo E, Castelli A, Fattizzo B, Beltrami G, Bocchia M, Molteni A, Fenaux P

Primary Author:

Oliva EN

Journal Title:

Journal of Clinical Oncology

Original Publication Date:

Oct 2023

Purpose: In

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)

Myelodysplastic syndromes with ring sideroblasts

Author(s):

Bruzzese A, Vigna E, Martino EA, Mendicino F, Lucia E, Olivito V, Bova C, Barbato A, Filippelli G, Capodanno I, Neri A, Morabito F, Gentile M

Primary Author:

Bruzzese A

Journal Title:

Hematological Oncology

Original Publication Date:

Oct 2023

Bone Marrow Disease(s):

Myelodysplastic Syndromes (MDS)

Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria

Author(s):

Schrezenmeier H, Kulasekararaj A, Mitchell L, de Latour RP, Devos T, Okamoto S, Wells R, Popoff E, Cheung A, Wang A, Tomazos I, Patel Y, Lee JW

Primary Author:

Schrezenmeier H

Journal Title:

Annals of Hematology

Original Publication Date:

Oct 2023

Bone Marrow Disease(s):

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia

Author(s):

Shinn LT, Benitez LL, Perissinotti AJ, Reid JH, Buhlinger KM, van Deventer H, Barth D, Wagner CB, Zacholski K, Desai R, Soule A, Stump SE, Weis TM, Bixby D, Burke P, Pettit K, Marini BL

Primary Author:

Shinn LT

Journal Title:

International Journal of Hematology

Original Publication Date:

Oct 2023

Eltrombopag has been shown to improve response rates when added to standard therapy in adults with severe

Bone Marrow Disease(s):

Aplastic Anemia