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Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

Author(s): 
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, Coates TD, Chawla A, Castillo P, Kurre P, Gamper C, Bennett CM, Joshi S, Geddis AE, Boklan J, Nalepa G, Rothman JA, Huang JN, Kupfer GM, Cada M, Glader B, Walkovich KJ, Thompson AA, Hanna R
Primary Author: 
Rogers ZR
Journal Title: 
Haematologica
Original Publication Date: 
Apr 2019

Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe

Bone Marrow Disease(s): 

Eltrombopag for refractory severe aplastic anemia: dosing, duration, long term outcomes and clonal evolution

Author(s): 
Winkler T, Fan X, Cooper J, Desmond R, Young DJ, Townsley DM, Scheinberg P, Grasmeder S, Larochelle A, Desierto M, Valdez J, Lotter J, Wu C, Shalhoub RN, Calvo KR, Young NS, Dunbar CE
Primary Author: 
Winkler T
Journal Title: 
Blood
Original Publication Date: 
Apr 2019

Eltrombopag received FDA approval for the treatment of refractory severe

Bone Marrow Disease(s): 

Getting personal with myelodysplastic syndromes: is now the right time?

Author(s): 
Chokr N, Pine AB, Bewersdorf JP, Shallis RM, Stahl M, Zeidan AM
Primary Author: 
Chokr N
Journal Title: 
Expert Review of Hematology
Original Publication Date: 
Apr 2019

Commonly used scoring systems rely on blood counts, histological and cytological examination of

Bone Marrow Disease(s): 

Use of Immunosuppressive therapy for management of myelodysplastic syndromes: a systematic review and meta-analysis

Author(s): 
Stahl M, Bewersdorf JP, Giri S, Wang R, Zeidan AM
Primary Author: 
Stahl M
Journal Title: 
Haemotologica
Original Publication Date: 
Apr 2019

Immunosuppressive therapy is one therapy option for treatment of patients with lower-risk

Bone Marrow Disease(s): 

Reprogramming identifies functionally distinct stages of clonal evolution in myelodysplastic syndromes

Author(s): 
Hsu J, Reilly A, Hayes BJ, Clough CA, Konnick EQ, Torok-Storb B, Gulsuner S, Wu D, Becker PS, Keel SB, Abkowitz JL, Doulatov S
Primary Author: 
Hsu J
Journal Title: 
Blood
Original Publication Date: 
Apr 2019

Myeloid neoplasms, including

Bone Marrow Disease(s): 

MDS with 5q deletion and rare cKIT positive mastocytosis: a diagnostic and therapeutic challenge

Author(s): 
Sanders DS, Fennell T, Chisti MM
Primary Author: 
Sanders DS
Journal Title: 
BMJ Case Reports
Original Publication Date: 
Apr 2019

A patient with a diagnosis of myelodysplastic syndrome (MDS) with isolated 5q deletion underwent repeat

Bone Marrow Disease(s): 

Clonal Hematopoiesis and therapy related MDS/AML

Author(s): 
Desai P, Roboz GJ
Primary Author: 
Desai P
Journal Title: 
Best Practice & Research. Clinical Heamatology
Original Publication Date: 
Mar 2019

Clonal Hematopoiesis is defined as the presence of mutations in peripheral blood in the absence of myeloid malignancies and is thought to occur as a normal part of ageing due to the fitness advantage conferred by these mutations in an ageing hematopoietic compartment.

Bone Marrow Disease(s): 

A phase 2 trial of the oral smoothened inhibitor glasdegib in refractory myelodysplastic syndromes (MDS)

Author(s): 
Sallman DA, Komrokji RS, Sweet KL, Mo Q, McGraw KL, Duong VH, Zhang L, Nardelli LA, Padron E, List AF, Lancet JE.
Primary Author: 
Sallman DA
Journal Title: 
Leukemia Research
Original Publication Date: 
Mar 2019

Hypomethylating agent (HMA) failure myelodysplastic syndrome (MDS) patients have poor outcomes and urgent need for novel therapies. Hedgehog pathway signaling upregulation plays a central role in myeloid neoplasm pathogenesis and leukemia stem cell survival. We evaluated the efficacy and safety of the smoothened inhibitor glasdegib in HMA-failure MDS (n = 35, median age 73 years). According to the

Bone Marrow Disease(s):