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Recurrent MYB rearrangement in blastic plasmacytoid dendritic cell neoplasm
TLDR
This work states thatymphoproliferative Disorders Team, Sorbonne Universités, UPMC Univ Paris 06, UMR_S 1138, Centre de Recherche des Cordeliers, Paris, France and Karyopharm Therapeutics, Newton, MA, USA contributed equally to this work. Expand
Integration Mapping of piggyBac-Mediated CD19 Chimeric Antigen Receptor T Cells Analyzed by Novel Tagmentation-Assisted PCR
TLDR
A new tagmentation-assisted PCR (tag-PCR) system is described that can determine the integration sites of transgenes without using restriction enzyme digestion and allows library preparation in fewer steps than with other methods. Expand
Choreito formula for BK virus-associated hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation.
TLDR
Choreito may be a safe and considerably promising therapy for the hemostasis of BKV-HC after HSCT. Expand
Successful treatment of pulmonary hypertension with beraprost and sildenafil after cord blood transplantation for infantile leukemia
TLDR
A case of severe PH that developed in an infant with acute leukemia following administration of busulfan as a preconditioner for cord blood transplantation is reported, which to the authors' knowledge is the first reported successful use of this regimen in PH following transplantation for infantile leukemia. Expand
Correlation of rabbit antithymocyte globulin serum levels and clinical outcomes in children who received hematopoietic stem cell transplantation from an alternative donor
TLDR
It is suggested that targeted rATG administration may protect patients from severe acute GVHD without increasing the risk of EBV reactivation or relapse. Expand
Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy
TLDR
Allogeneic SCT with reduced intensity conditioning for patients with ALD was safely performed without major transplant-related complications even in symptomatic patients and neurological symptoms were stabilized after SCT in patients without internal capsule involvement. Expand
Genetic Background of Idiopathic Bone Marrow Failure Syndromes in Children
TLDR
The genetic background of childhood AA/RCC/RCMD was targeted to characterize and support the diagnosis of dyskeratosis congenita, a severe decrease in bone marrow cellularity in children with inherited bone marrow failure syndromes. Expand
Paroxysmal nocturnal hemoglobinuria and telomere length predicts response to immunosuppressive therapy in pediatric aplastic anemia
TLDR
The combined absence of a minor paroxysmal nocturnal hemoglobinuria population and a short telomere length is an efficient predictor of poor immunosuppressive therapy response, which should be considered while deciding treatment options: immunosppressive therapy or first-line hematopoietic stem cell transplantation. Expand
MEF2D-BCL9 Fusion Gene Is Associated With High-Risk Acute B-Cell Precursor Lymphoblastic Leukemia in Adolescents.
TLDR
A novel MEF2D-BCL9 fusion is identified that characterizes a novel subset of pediatric ALL, predicts poor prognosis, and may be a candidate for novel molecular targeting. Expand
Clinical utility of next‐generation sequencing‐based minimal residual disease in paediatric B‐cell acute lymphoblastic leukaemia
TLDR
The study suggests the promising potential of NGS‐MRD for patients with B‐cell ALL, and exhibited statistically significant associations with inferior leukaemia‐free survival. Expand
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