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A Journey from Blood Cells to Genes and Back

Author(s): 
Luzzatto L
Primary Author: 
Luzzatto L
Journal Title: 
Annual Review of Genomics and Human Genetics
Original Publication Date: 
Aug 2023

I was attracted to hematology because by combining clinical findings with the use of a microscope and simple laboratory tests, one could often make a diagnosis. I was attracted to genetics when I learned about inherited blood disorders, at a time when we had only hints that somatic mutations were also important. It seemed clear that if we understood not only what genetic changes caused what diseases but also the mechanisms through which those genetic changes contribute to cause disease, we could improve management.

Bone Marrow Disease(s): 

Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria

Author(s): 
Kulasekararaj AG, Lehtinen AE, Forsyth C, Gandhi S, Griffin M, Körper S, Mikala G, Muus P, Overgaard U, Patriquin CJ, Pullon H, Shen YM, Spearing R, Szer J, De la Borderie G, Duda PW, Farzaneh-Far R, Ragunathan S, Sayegh CE, Vadysirisack DD
Primary Author: 
Kulasekararaj AG
Journal Title: 
Haematologica
Original Publication Date: 
Aug 2023
Bone Marrow Disease(s): 

March for Marrow California 2024

March for Marrow LA 2024 is back! Welcome to our second in-person March for Marrow LA, since 2019! Our 14th Annual March for Marrow will be held at the iconic Shoreline Village, situated along the beautiful Long Beach Marina at 429 Shoreline Village Drive, Long Beach, CA. Following two years of virtual events, we invite everyone in California to be with us in-person or virtually as we celebrate being together. We hope to connect with patients and families from all parts of the great state of California.

Event Date: 
Sat, 04/27/2024 - 7:00am (EDT)
Community Event Type: 

March for Marrow Texas 2024

March for Marrow TX 2023 is back! Welcome to our second in-person March for Marrow TX, The Woodlands, since 2019! Our 14th Annual March for Marrow will be held at the beautiful Northshore Park, 2505 Lake Woodlands Dr, The Woodlands, TX. Following three years of virtual events, we invite everyone across the state of Texas to be with us in-person or virtually as we celebrate being together. We will stand with our arms outstretched from Houston to El Paso and from Amarillo to Brownsville, from east to west and north to south.

Event Date: 
Sat, 04/06/2024 - 8:00am (EDT)
Community Event Type: 

Why do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS

Author(s): 
Frumm SM, Shimony S, Stone RM, DeAngelo DJ, Bewersdorf JP, Zeidan AM, Stahl M
Primary Author: 
Frumm SM
Journal Title: 
Blood Advances
Original Publication Date: 
Jul 2023

Approval of new agents to treat higher risk (HR) myelodysplastic syndrome (MDS) has stalled since the approval of DNA methyltransferase inhibitors (DNMTi). In addition, the options for patients with lower risk (LR) MDS who have high transfusion needs and do not harbor ring sideroblasts or 5q- syndrome are limited. Here, we review the current treatment landscape in MDS and identify areas of unmet need, such as treatment after failure of erythropoiesis-stimulating agents or DNMTis, TP53-mutated disease, and MDS with potentially targetable mutations.

Bone Marrow Disease(s): 

Transplantation Referral Patterns for Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia at Academic and Community Sites in the Connect® Myeloid Disease Registry: Potential Barriers to Care

Author(s): 
Tomlinson B, de Lima M, Cogle CR, Thompson MA, Grinblatt DL, Pollyea DA, Komrokji RS, Roboz GJ, Savona MR, Sekeres MA, Abedi M, Garcia-Manero G, Kurtin SE, Maciejewski JP, Patel JL, Revicki DA, George TI, Flick ED, Kiselev P, Louis CU, DeGutis IS
Primary Author: 
Tomlinson B
Journal Title: 
Transplantation and Cellular Therapy
Original Publication Date: 
Jul 2023

Hematopoietic stem cell transplantation (HCT) is indicated for patients with higher-risk (HR)

Bone Marrow Disease(s):