Very long-term follow-up of aplastic anemia treated with immunosuppressive therapy or allogeneic hematopoietic cell transplantation

ConclusionVery long term survivors after AA are those with stable hematopoietic recovery.
Source: Annals of Hematology - Category: Hematology Source Type: research

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Authors: Chen X, Yu X, Wang DD, Xu H, Li Z Abstract Bone marrow transplants (BMT) are an established therapeutic strategy for patients with severe aplastic anemia, acute lymphoblastic leukemia, acute myeloid leukemia or chronic myeloid leukemia. However, the successful application of BMT is limited by graft-vs.-host disease (GVHD). Ciclosporin has been widely used for treating GVHD in pediatric patients who underwent BMT. The present study aimed to optimize the dosage of ciclosporin for safety and effectiveness based on population pharmacokinetics. A non-linear mixed-effects model was used to analyze the clinical d...
Source: Experimental and Therapeutic Medicine - Category: General Medicine Tags: Exp Ther Med Source Type: research
Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder characterized by bone marrow failure (BMF; cytopenia or aplastic anemia), exocrine pancreatic insufficiency, and skeletal abnormalities, with a predisposition to myelodysplasia (MDS) and acute myeloid leukemia (AML)1. Although most individuals with SDS have some hematologic manifestations of their disease, the majority of them do not require transplantation. Hematopoietic stem cell transplantation (HSCT), however, remains the only curative therapy for 19-36 % of patients with SDS who develop BMF or transformation to myeloid malignancy.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder characterized by bone marrow failure (BMF; cytopenia or aplastic anemia), exocrine pancreatic insufficiency, and skeletal abnormalities, with a predisposition to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) [1]. Although most individuals with SDS have some hematologic manifestations of their disease, the majority do not require transplantation. However, hematopoietic stem cell transplantation (HSCT) remains the sole curative therapy for 19% to 36% of patients with SDS who develop BMF or transformation to myeloid malignancy.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder characterized by bone marrow failure (BMF; cytopenia or aplastic anemia), exocrine pancreatic insufficiency, and skeletal abnormalities, with a predisposition to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) [1]. Although most individuals with SDS have some hematologic manifestations of their disease, the majority do not require transplantation. However, hematopoietic stem cell transplantation (HSCT) remains the sole curative therapy for 19% to 36% of patients with SDS who develop BMF or transformation to myeloid malignancy.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: Pediatric Source Type: research
Genetic abnormalities can predispose patients to develop acute myeloid leukemia (AML), aplastic anemia (AA) and myelodysplastic syndrome (MDS). These mutations are more often seen in pediatric patients so genetic screening is more routinely performed in that population. Recent algorithms propose screening in adolescent and young adults (AYAs) with AML/AA/MDS. However, this is not routine practice in community hospitals where most AYAs are treated. Discovery of familial genetic abnormalities can impact donor choice in patients undergoing allogeneic stem cell transplant and treatment/screening in family members.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Tags: 145 Source Type: research
ConclusionWe suggest a one ‐compartment population model with first‐order elimination to capture the pharmacokinetic profile for basiliximab for this patient population.
Source: Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy - Category: Drugs & Pharmacology Authors: Tags: ORIGINAL RESEARCH ARTICLE Source Type: research
stic Anemia Working Party of the European Society for Blood and Marrow Transplantation Abstract This follow-up study of the randomized prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving Antithymoglobulin and Cyclosporine, with and without G-CSF. We aimed to evaluate the long-term effect of G-CSF on overall survival, event-free survival, probability of secondary myelodysplastic syndrome or acute myeloid leukemia, clinical paroxysmal nocturnal hemoglobinuria, relapse, avascular osteonecrosis and chronic kidney disease. The median follow-up was 11.7 years (10.9-12.5). Overal...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Allogeneic HLA-matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) for adults with acquired severe aplastic anemia (SAA) is currently recommended after failure to respond to a course of immunosuppressive therapy (IST) 1,2. This guidance reflects the historical developments in HSCT and IST, and the improving outcomes of MUD HSCT over time. Although SAA patients are surviving long term after both HSCT and IST, the long-term complications of clonal transformation to myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) and hemolytic PNH after IST in up to 15-26% of patients at 10 years are notable 3-7.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Allogeneic HLA-matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) for adults with acquired severe aplastic anemia (SAA) is currently recommended after failure to respond to a course of immunosuppressive therapy (IST) [1,2]. This guidance reflects the historical developments in HSCT and IST, as well as the improving outcomes of MUD HSCT over time. Although patients with SAA are surviving long term after both HSCT and IST, the long-term complications of clonal transformation to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) and hemolytic paroxysmal nocturnal hemoglobinuria (PNH)...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
This study may open up new potential therapeutic avenues for the treatment of patients with chronic infection, inflammatory diseases, and cancer.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 506. Hematopoiesis and Stem Cells: Microenvironment, Cell Adhesion, and Stromal Stem Cells Source Type: research
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