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A Phase I Trial of SYK Inhibition with Fostamatinib in the Prevention and Treatment of Chronic Graft-Versus-Host Disease
Chronic graft-versus-host disease (cGVHD) is the most common cause of late morbidity and non-relapse mortality among patients who have undergone allogeneic hematopoietic stem cell transplantation (HCT).1 The presentation of cGVHD is highly heterogeneous and may involve multiple organ systems, often leading to detrimental impacts on quality of life and functional status.2,3 Standard first-line treatment involves prolonged systemic steroids, which can be complicated by numerous steroid-related toxicities including hyperglycemia, myopathy, and osteoporosis.
Source: Biology of Blood and Marrow Transplantation - December 24, 2022 Category: Hematology Authors: Chenyu Lin, Rachel A. DiCioccio, Tarek Haykal, William C. McManigle, Zhiguo Li, Sarah M. Anand, Jonathan C. Poe, Sonali J. Bracken, Wei Jia, Edwin P. Alyea, Adela R. Cardones, Taewoong Choi, Cristina Gasparetto, Michael R. Grunwald, Therese Hennig, Yubin Tags: AMA Source Type: research

Neurocognitive and Physical Functioning Screening in Hematopoietic Cell Transplant (HCT) Survivorship Clinic
Neurocognitive impairment and musculoskeletal complications are among the most common complications of long-term allogeneic hematopoietic cell transplant (HCT) survivors, for which recommendations regarding routine screening and therapeutic interventions remain limited. Changes in cognitive functioning have been described in 40% of long-term allogeneic hematopoietic cell transplant survivors, and although the incidence of musculoskeletal complications has not been clearly defined, is very common- ranging from steroid-induced myopathy, myositis related to chronic graft-versus-host disease (GVHD), and fasciitis/scleroderma manifestations.
Source: Biology of Blood and Marrow Transplantation - January 24, 2020 Category: Hematology Authors: Christina S Ferraro, Clarence Williams, Beth Dixon, Laura Bernhard, Navneet S. Majhail, Betty K. Hamilton Tags: 563 Source Type: research

Outcomes of Autologous Hematopoietic Stem Cell Transplant in Sporadic Late Onset Nemaline Myopathy with Associated Monoclonal Gammopathy of Unknown Significance
Background: Sporadic Late Onset Nemaline Myopathy (SLONM) is a rare muscle disorder with subacute progression, characterized by axial and proximal muscle weakness and atrophy, and the presence of nemaline rods in myofibers. When SLONM is associated with a monoclonal gammopathy of undetermined significance (SLONM/MGUS), patients seem to fare worse compared to patients with SLONM and no MGUS. Supportive and immunosuppressive therapies have yielded poor outcomes for most patients with SLONM/MGUS; IVIG have been beneficial in some.
Source: Biology of Blood and Marrow Transplantation - February 1, 2018 Category: Hematology Authors: Ankit J. Kansagra, Francis Buadi, Margherita Milone, Elie Naddaf, Morie Gertz, Angela Dispenzieri, Shaji Kumar, David Dingli, Martha Q. Lacy, Prashant Kapoor, Wilson I. Gonsalves, Rahma Warsame, Eli Muchtar, Robert Kyle, Taxiarchis Kourelis Source Type: research