Andrew Charles Dietz

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA

Jenny Anne Downs, Dominique C. Pichard, Walter E. Kaufmann, Joseph P. Horrigan et autres

INTRODUCTION: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what …

au, us, gb (code pays fourni par la source)

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

Christine Duncan, Jacob R. Bledsoe, Bartosz J. Grzywacz, Amy Beckman et autres

Background Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenoleukodystrophy. However, the risk of oncogenesis with eli-cel is unclear. …

us (code pays fourni par la source)

Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

Florian Eichler, Christine Duncan, Patricia Leonor Musolino, Troy C. Lund et autres

BACKGROUND: complementary DNA, is being tested in persons with cerebral adrenoleukodystrophy. METHODS: In a phase 2-3 study, we evaluated the efficacy and safety of eli-cel therapy in boys with early-stage cerebral adrenoleukodystrophy and evidence of active inflammation on magnetic resonance imaging (MRI). …

fr, us, gb (code pays fourni par la source)

Recommendations for surveillance of pulmonary dysfunction among childhood, adolescent, and young adult cancer survivors: a report from the International Late Effects of Childhood Cancer Guideline Harmonization Group

Maria Otth, Rahel Kasteler, Renée L. Mulder, Jennifer Elise Agrusa et autres

Childhood, adolescent, and young adult (CAYA) cancer survivors are at risk of pulmonary dysfunction. Current follow-up care guidelines are discordant. Therefore, the International Late Effects of Childhood Cancer Guideline Harmonization Group established and convened a panel of 33 experts to develop evidence-based …

ch, nl, us, il, gb, de, fr (code pays fourni par la source)

Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adrenoleukodystrophy (CALD)

Christine Duncan, Paul J. Orchard, Florian Eichler, Jörn‐Sven Kühl et autres

Eli-cel, an autologous hematopoietic stem cell (HSC) gene therapy consisting of CD34+ cells transduced with ABCD1 cDNA using a Lenti-D lentiviral vector (LVV), is approved for the treatment of CALD. To describe conditioning and engraftment data from patients with CALD treated with …

us, de, gb, fr, it, au, br, ar, nl (code pays fourni par la source)

Recommendations for surveillance of pulmonary dysfunction among childhood, adolescent, and young adult cancer survivors: a report from the International Late Effects of Childhood Cancer Guideline Harmonization Group

Maria Otth, Rahel Kasteler, Renée L. Mulder, Jennifer Elise Agrusa et autres

ABSTRACT Childhood, adolescent, and young adult (CAYA) cancer survivors are at risk of pulmonary dysfunction. Current follow-up care guidelines are discordant. Therefore, the International Late Effects of Childhood Cancer Guideline Harmonization Group established and convened a panel of 33 experts to develop …

ch, nl, us, il, gb, de, fr (code pays fourni par la source)

Data from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

Abstract Background: Childhood cancer survivors (CCS) are more insulin resistant and have higher levels of several cardiovascular risk factors even while still children. This study examines specific treatment exposures associated with cardiovascular risk factors and insulin resistance. Methods: CCS of ages 9 …

Data from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

Abstract Background: Childhood cancer survivors (CCS) are more insulin resistant and have higher levels of several cardiovascular risk factors even while still children. This study examines specific treatment exposures associated with cardiovascular risk factors and insulin resistance. Methods: CCS of ages 9 …

Supplementary Tables 1 and 2 from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

PDF - 55KB, Supplementary Table S1. Number (N) and percent (%) of therapeutic exposures used in the development of the tree model within each final treatment combination category. Supplementary Table S2: Cumulative dose categories of chemotherapy agents.

Supplementary Figure 1 from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

PDF - 26KB, Consort Diagram of study participants.

Supplementary Tables 1 and 2 from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

PDF - 55KB, Supplementary Table S1. Number (N) and percent (%) of therapeutic exposures used in the development of the tree model within each final treatment combination category. Supplementary Table S2: Cumulative dose categories of chemotherapy agents.

Supplementary Figure 1 from Impact of Treatment Exposures on Cardiovascular Risk and Insulin Resistance in Childhood Cancer Survivors

K. Scott Baker, Eric Jessen Chow, Pamela J. Goodman, Wendy M. Leisenring et autres

PDF - 26KB, Consort Diagram of study participants.