Bone Marrow Failure Disorders

01/01/2023 - "Androgens have been reported to elongate telomeres in retrospective and prospective trials with patients with telomeropathies, mainly with bone marrow failure. "
05/18/2023 - "Androgens have represented the historical therapeutic backbone of bone marrow failure (BMF) syndromes. "
01/01/2020 - "Androgens have been used to improve blood counts in different bone marrow failure conditions. "
12/01/2018 - "Androgens are a therapeutic option for DC/TBD-associated bone marrow failure (BMF). "
09/10/2009 - "Androgens have been used in the treatment of bone marrow failure syndromes without a clear understanding of their mechanism of action. "

08/01/1989 - "Oral cyclosporin-A is effective treatment for untreated and also for previously immunosuppressed patients with severe bone marrow failure."
01/01/2023 - "An immune-mediated bone marrow failure mice model was established and forty C57BL/6 mice were randomly assigned into 4 groups: normal group, model group,cyclosporine (CsA) group and QG group, with 10 mice in each group.The"
12/01/2002 - "Taken together, we provide evidence for clonal T-cell mediated bone marrow failure with cyclic impairment of thrombopoiesis responsive to cyclosporine therapy."
11/01/2006 - "To our knowledge, this is the first case report of cyclosporine used for treatment of bone marrow failure in a patient with acute intermittent porphyria. "
11/01/2006 - "We describe a patient with acute intermittent porphyria who developed bone marrow failure and was successfully treated with cyclosporine. "

01/01/2020 - "[Efficacy of thrombopoietin in bone marrow failure]."
01/01/2017 - "Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin."
09/01/2011 - "Moreover, although mice functionally compromised for either Erg or Mpl, the receptor for thrombopoietin, a key regulator of HSC quiescence, maintained sufficient HSC activity to sustain hematopoiesis, Mpl(-/-) Erg(+/Mld2) compound mutant mice displayed exacerbated stem cell deficiencies and bone marrow failure. "
10/01/2016 - "Hypomegakaryocytic thrombocytopenia (HMT): an immune-mediated bone marrow failure characterized by an increased number of PNH-phenotype cells and high plasma thrombopoietin levels."
06/01/2013 - "In conclusion, increased plasma thrombopoietin levels were associated with a favorable prognosis of bone marrow failure and could, therefore, represent a reliable marker for a benign subset of myelodysplastic syndrome."

04/01/2012 - "This article proposes assessment of patients with MDS and related bone marrow failure syndromes to determine suitability for iron chelation. "
07/01/2008 - "Many patients with bone marrow failure syndromes need frequent transfusions of red blood cells, and most of them eventually suffer from organ dysfunction induced by excessively accumulated iron. "
03/01/2008 - "Many patients with bone marrow failure need repetitive transfusion of red blood cells and most of them finally suffer from organ dysfunction induced by excessively accumulated iron. "
04/01/2012 - "When should iron chelation therapy be considered in patients with myelodysplasia and other bone marrow failure syndromes with iron overload?"
01/01/2022 - "Long-term eltrombopag for bone marrow failure depletes iron."

05/01/2014 - "In addition, although danazol can sometimes be effective for treating bone marrow failure, hepatotoxicity can be a major complication. "
12/01/1996 - "In our experience, danazol appears effective and safe in the subset of patients with AMM whose disease is mainly characterized by bone-marrow failure. "
12/02/2016 - "Danazol, which is traditionally less preferred for treating cytopenias, is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulting from telomere disease."
02/15/2012 - "Treatment of the bone marrow failure in Fanconi anemia patients with danazol."
01/01/2018 - "Treatment modalities included lung transplantation for IIP (n=5 [29%]), with 3 patients developing signs of acute cellular rejection (2, grade A2; 1, grade A1); danazol therapy for cytopenias (n=4 [24%]), with only 1 out of 4 patients showing a partial hematologic response; and allogeneic hematopoietic stem cell transplant for progressive bone marrow failure (n=2), with 1 patient dying from transplant-related complications. "

03/01/2008 - "Oral iron chelator(deferasirox) developed for the treatment of transfusional iron overload is also very useful for the management of patients with bone marrow failure syndromes."
12/01/2018 - "Eltrombopag (EP), a small-molecule thrombopoietin receptor (TPO-R) agonist and potent intracellular iron chelator, has shown remarkable efficacy in stimulating sustained multilineage hematopoiesis in patients with bone marrow failure syndromes, suggesting an effect at the most immature hematopoietic stem and multipotent progenitor level. "

04/01/1992 - "These results indicate that treatment with GM-CSF can be an effective biological monotherapy for radiation-induced bone marrow failure, but that for higher radiation doses the number of GM-CSF responsive target cells will become a critical determinant of therapeutic efficacy."
12/01/2003 - "Sequential IL-3/GM-CSF effectively raised blood counts in some patients with bone marrow failure at doses that were tolerated well. "
01/01/1993 - "GM-CSF as primary treatment for myelodysplasia and other types of bone marrow failure is also of interest. "
07/01/1990 - "Effects of granulocyte-macrophage colony-stimulating factor in iatrogenic myelosuppression, bone marrow failure, and regulation of host defense."
12/01/2003 - "Because of the paucity of effective therapies for patients with chronic bone marrow failure states, the authors studied the biologic activity of sequential IL-3/GM-CSF in such patients. "

05/01/1990 - "The findings may have therapeutic implications, as combinations of growth factors may be more effective in treating bone marrow failure syndromes."
07/01/2007 - "Hematopoietic growth factors in the treatment of acquired bone marrow failure states."
07/01/2007 - "The clinical availability of recombinant hematopoietic growth factors was initially thought to be breakthrough in the treatment of bone marrow failure syndromes. "
12/01/2003 - "These results indicate that early-acting growth factors can induce durable, multilineage responses in a subset of individuals with bone marrow failure."
05/01/1998 - "This article reviews the clinical use of old and new hematopoietic growth factors in acquired and inherited bone marrow failure, and discusses emerging concerns about long term toxicity of these factors."

05/01/1998 - "Granulocyte colony-stimulating factor (G-CSF) has been shown to improve the neutropenic status of patients with bone marrow failure. "
04/01/2003 - "Patient C also developed bone marrow failure and was treated with granulocyte colony-stimulating factor as well as supportive care. "
01/01/2021 - "Here we present a case of a 28-year-old male who presented with sero-negative hepatitis and rapidly progressive bone marrow failure who was given a trial of Granulocyte Colony Stimulating Factor followed by a successful allogenic bone marrow transplant."
07/01/2009 - "Using a murine model of Fanconi anemia (Fanca(-/-) mice), we found that the Fanca deficiency was associated with a profound defect in hematopoietic stem cells and progenitors mobilization in response to G-CSF in absence of bone marrow failure, which correlates with the findings of clinical trials in Fanconi anemia patients. "
01/01/2019 - "We detected CSF3R mutations in CN patients with different genetic backgrounds, but not in patients with other types of bone marrow failure syndromes chronically treated with G-CSF (e.g., Shwachman-Diamond Syndrome). "

07/01/1998 - "We report on two female patients with Hodgkin's disease (HD) followed between 1970 and 1995 at the institute of Hematology and Medical Oncology "Seràgnoli" in Bologna, who developed ITP unrelated to bone marrow failure, 26 and 15 months after achievement of complete hematologic remission from HD."
07/01/2023 - "Reticulated platelet count may be a useful diagnostic tool to differentiate between ITP and thrombocytopenia caused by bone marrow failure. "
07/01/2023 - "Patients with ITP had a significantly higher median IPF% (median=26.65, IQR=15-39.4) than thrombocytopenia due to bone marrow failure (median=9.25, IQR=4.55-14.30) and healthy controls (median=7, IQR=4.40-9.90), with a p-value of 0.001. "
01/01/2020 - "Immune thrombocytopenic purpura (ITP) is an autoimmune thrombocytopenia with shortened platelet survival and relative bone marrow failure. "
01/01/2015 - "Our data show that acute and chronic ITP may occur in FA patients and impose individual diagnostic and therapeutic challenges in this rare congenital bone marrow failure/tumor predisposition syndrome. "

12/01/1988 - "Thus, CSFs have considerable promise for therapy for bone marrow failure states."
10/26/2020 - "However, these therapies are only effective in 50% of patients, and many develop resistance to therapy, often resulting in bone marrow failure or leukemic transformation. "
01/01/2020 - "To some extent, this study had shown a relatively better synergistic effect in murine models of immune-mediated bone marrow failure after the combination therapy of Rapa and Elt, which was a promising clinical utility in SAA treatment."
02/15/2005 - "Herein, we provide an overview of the clinical research investigations of thrombopoietic factors in chronic bone marrow failure, and the emerging insights these studies provide for understanding the process of thrombopoiesis and its therapy in this setting."
12/01/2003 - "Recent studies have suggested that ex vivo expansion of autologous hematopoietic cells could be a therapy of choice for the treatment of bone marrow failure. "

01/01/2015 - "Bone marrow failure in DC can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). "
10/01/2023 - "Haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Patients with Acquired Hypocellular Bone Marrow Failure."
04/01/2023 - "Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for progressive bone marrow failure. "
12/09/2022 - "While several nontransplant treatments are currently under evaluation, hematopoietic stem cell transplantation (HSCT) remains the only therapeutic option for bone marrow failure (BMF). "
02/04/2022 - "There is no specific treatment for PS, which remains supportive care according to symptoms; however, hematopoietic stem cell transplantation may be considered in cases of bone marrow failure.We"

03/01/2005 - "With marked improvement in rates of survival after allogeneic hematopoietic cell transplantation for selected congenital bone marrow failure disorders, emphasis is now being placed on improving quality of life and reducing late effects. "
01/01/2021 - "Twenty-six had bone marrow failure and 12 underwent haematopoietic cell transplantation. "
01/01/2020 - "Fanconi anaemia (FA) is a rare disorder characterized by progressive bone marrow failure that requires haematopoietic cell transplantation (HCT). "
03/01/2005 - "Hematopoietic cell transplantation for congenital bone marrow failure."
01/01/2024 - "Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative therapeutic treatment for patients with high-risk hematologic malignancies and bone marrow failure syndromes. "

01/01/2018 - "We found no evidence to determine the safety and efficacy of therapeutic platelet transfusion compared with prophylactic platelet transfusion for people with long-term bone marrow failure disorders. "
02/01/1988 - "To determine the relative importance of clinical factors on the efficacy of platelet transfusions, 941 pooled platelet transfusions from HLA-unmatched donors were studied prospectively in 133 patients with bone marrow failure. "
01/01/2018 - "People with bone marrow failure depend on long-term platelet transfusion support, but the only trial that assessed a therapeutic strategy was halted. "
01/01/2018 - "We included all people with long-term bone marrow failure disorders that require platelet transfusions, including neonates. "
01/01/2018 - "Comparison of a therapeutic-only versus prophylactic platelet transfusion policy for people with congenital or acquired bone marrow failure disorders."

05/01/2015 - "CSNK1A1 resides on the critically deleted region, and deletion of this gene has been shown in mouse knockout and transplantation studies to produce some characteristics of bone marrow failure, including a proliferative advantage. "
01/03/2014 - "This study investigated BMSCs homing in mice transplantation models after bone marrow failure. "
08/25/2023 - "Outcome of first or second transplantation using unrelated umbilical cord blood without ATG conditioning regimen for pediatric bone marrow failure disorders."
01/01/2023 - "Those infants with MECOM-associated bone marrow failure require HSC transplantation for survival. "
01/01/2022 - "Adult-onset cases must be recognized, and transplantation should be performed during bone marrow failure."