Efficacy and safety of avatrombopag in combination with immunosuppressive therapy in treatment-naïve and relapsed/refractory severe aplastic anaemia: protocol for the DIAAMOND-Ava-FIRST and DIAAMOND-Ava-NEXT Bayesian Optimal Phase II trials
Introduction
Immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and ciclosporin is standard of care for patients with severe aplastic anaemia (sAA) not eligible or suitable for allogeneic stem cell transplant. While patients respond to IST, few achieve complete responses and a significant proportion are refractory or relapse. The addition of eltrombopag, a thrombopoietin-receptor agonist (TPO-A), to IST has been shown to improve haematological responses in sAA. Avatrombopag is a second-generation TPO-A with potential advantages over eltrombopag. However, to date avatrombopag has not been studied in sAA.
Met...
Source: BMJ Open - January 18, 2024 Category: General Medicine Authors: McQuilten, Z., Heritier, S., Fox, L., Fox, V., Young, L., Blombery, P., Cunningham, I., Curnow, J., Higgins, A., Hiwase, D. K., Filshie, R., Firkin, F., Lacaze, P., Mason, K., Mills, A. K., Pepperell, D., Patil, S., Stevenson, W., Szer, J., Waters, N., Wi Tags: Open access, Haematology (incl blood transfusion) Source Type: research
Case report: Immune pressure on hematopoietic stem cells can drastically expand glycosylphosphatidylinositol-deficient clones in paroxysmal nocturnal hemoglobinuria
ConclusionsThe PNH clones that had increased excessively before BMT decreased, but persisted at low percentages for more than four years after the immunoablative conditioning regimen followed by syngeneic BMT. These findings indicate that as opposed to conventional theory, immune pressure on HSCs, which caused BM failure before BMT, was sufficient for PIGA-mutated HSCs to clonally expand to develop PNH. (Source: Frontiers in Immunology)
Source: Frontiers in Immunology - January 15, 2024 Category: Allergy & Immunology Source Type: research
Impact of center volume on chronic graft versus host disease in patients with allogeneic stem cell transplantation
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment option for various hematological malignancies and benign conditions, such as aplastic anemia.1,2 However, owing to its specialized and complex nature, allogeneic HSCT practice may vary among transplant centers, including variations in patient selection criteria, transplantation regimens, supportive care practices, and management of post-transplant complications.3 –7 These variations may impact transplant outcomes, making the center experience a crucial factor in improving patient outcomes. (Source: Biology of Blood and Marrow Transplantation)
Source: Biology of Blood and Marrow Transplantation - January 10, 2024 Category: Hematology Authors: Yoshimitsu Shimomura, Tetsuhisa Kitamura, Makoto Murata, Keitaro Matsuo, Yuri Ito, Tatsuo Ichinohe, Yoshiko Hashii, Hideki Goto, Koji Kato, Fumihiko Ishimaru, Atsushi Sato, Makoto Onizuka, Atsumi Yanagisawa, Marie Ohbiki, Ken Tabuchi, Yoshiko Atsuta, Taka Source Type: research
A multicentre ambispective observational study into the incidence and clinical management of aplastic anaemia in Spain (IMAS study)
In conclusion, the incidence, characteristics and management of AA in our study are consis tent with that reported previously. In terms of survival, although the global long-term OS rate was good, there is room for improvement, particularly in older patients. Finally, what appears to be a worse long-term survival of MAA patients, as reported previously, reinforces the importance of not un derestimating this condition when diagnosed as MAA. (Source: Annals of Hematology)
Source: Annals of Hematology - January 4, 2024 Category: Hematology Source Type: research
High- vs regular-dose recombinant human thrombopoietin plus cyclosporine A in patients with newly diagnosed non-severe aplastic anemia: a retrospective cohort study
CONCLUSIONS: Adding a higher dose of rhTPO can further accelerate platelet recovery and platelet transfusion independence in patients with newly diagnosed NSAA.PMID:38156735 | DOI:10.1080/16078454.2023.2298523 (Source: Hematology)
Source: Hematology - December 29, 2023 Category: Hematology Authors: Yuan Yang Qinglin Hu Chen Yang Miao Chen Bing Han Source Type: research
All Roads Lead to Interferon- γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
Medicina (Kaunas). 2023 Dec 14;59(12):2170. doi: 10.3390/medicina59122170.ABSTRACTBone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T...
Source: Medicina (Kaunas) - December 23, 2023 Category: Universities & Medical Training Authors: Bianca Serio Valentina Giudice Carmine Selleri Source Type: research
All Roads Lead to Interferon- γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
Medicina (Kaunas). 2023 Dec 14;59(12):2170. doi: 10.3390/medicina59122170.ABSTRACTBone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T...
Source: Medicina (Kaunas) - December 23, 2023 Category: Universities & Medical Training Authors: Bianca Serio Valentina Giudice Carmine Selleri Source Type: research
All Roads Lead to Interferon- γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
Medicina (Kaunas). 2023 Dec 14;59(12):2170. doi: 10.3390/medicina59122170.ABSTRACTBone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T...
Source: Medicina (Kaunas) - December 23, 2023 Category: Universities & Medical Training Authors: Bianca Serio Valentina Giudice Carmine Selleri Source Type: research
All Roads Lead to Interferon- γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
Medicina (Kaunas). 2023 Dec 14;59(12):2170. doi: 10.3390/medicina59122170.ABSTRACTBone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T...
Source: Medicina (Kaunas) - December 23, 2023 Category: Universities & Medical Training Authors: Bianca Serio Valentina Giudice Carmine Selleri Source Type: research
All Roads Lead to Interferon- γ: From Known to Untraveled Pathways in Acquired Aplastic Anemia
Medicina (Kaunas). 2023 Dec 14;59(12):2170. doi: 10.3390/medicina59122170.ABSTRACTBone marrow failure (BMF) syndromes are a heterogeneous group of benign hematological conditions with common clinical features including reduced bone marrow cellularity and peripheral blood cytopenias. Acquired aplastic anemia (AA) is caused by T helper(Th)1-mediated immune responses and cytotoxic CD8+ T cell-mediated autologous immune attacks against hematopoietic stem and progenitor cells (HSPCs). Interferon-γ (IFNγ), tumor necrosis factor-α, and Fas-ligand are historically linked to AA pathogenesis because they drive Th1 and cytotoxic T...
Source: Medicina (Kaunas) - December 23, 2023 Category: Universities & Medical Training Authors: Bianca Serio Valentina Giudice Carmine Selleri Source Type: research
