Share This Author
National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report.
This consensus document standardizes the criteria for diagnosis of chronic graft-versus-host disease (GVHD) and proposes a new clinical scoring system (0-3) that describes the extent and severity of chronic GVHD for each organ or site at any given time, taking functional impact into account.
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.
Diagnosis and management of paroxysmal nocturnal hemoglobinuria.
The primary clinical manifestations of paroxysmal nocturnal hemoglobinuria (PNH) are hemolytic anemia, marrow failure, and thrombophilia. However, PNH is not a simple binary diagnosis and both flow…
Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling.
It is necessary to select patients suitable for vaginal or laparoscopic mesh placement for Fanconi's anemia preoperatively on the basis of prior history and once they provide informed consent for surgery.
Risk of lymphoproliferative disorders after bone marrow transplantation: a multi-institutional study.
It is concluded that factors associated with altered immunity and T-cell regulatory mechanisms are predictors of both early- and late-onset PTLD.
Risk factors for lymphoproliferative disorders after allogeneic hematopoietic cell transplantation.
These findings identify subgroups of patients who underwent allogeneic HCT at elevated risk of PTLDs for whom prospective monitoring of Epstein-Barr virus activation and early treatment intervention may be particularly beneficial.
Solid cancers after bone marrow transplantation.
The trend toward an increased risk over time after transplantation and the greater risk among younger patients indicate the need for life-long surveillance.
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria.
Eculizumab is an effective therapy for PNH and Clinically significant improvements were found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire.
Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease.
Ruxolitinib therapy led to significant improvements in efficacy outcomes, with a higher incidence of thrombocytopenia, the most frequent toxic effect, than that observed with control therapy.
Long-term survival and late deaths after allogeneic hematopoietic cell transplantation.
The prospect for long-term survival is excellent for 2-year survivors of allogeneic HCT, however, life expectancy remains lower than expected.