4.7 Review

Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Hematology

Granulocyte Colony-Stimulating Factor Is Safe and Well Tolerated following Allogeneic Transplantation in Patients with Sickle Cell Disease

Niketa C. Shah et al.

Summary: This study analyzed the clinical outcomes of 62 sickle cell disease (SCD) patients receiving G-CSF treatment. The results showed that G-CSF does not adversely affect SCD HSCT recipients and can be safely used to enhance neutrophil recovery after transplantation.

TRANSPLANTATION AND CELLULAR THERAPY (2022)

Article Pediatrics

Allogeneic hematopoietic stem cell transplantation from sibling and unrelated donors in pediatric patients with sickle cell disease-A single center experience

Friederike Kogel et al.

Summary: HSCT is curative in SCD, but lack of HLA-identical sibling donors limits access for 70% of patients. Transplantations from HLA-matched unrelated donors have lower success rates, but show excellent overall and disease-free survival in small patient cohorts.

PEDIATRIC TRANSPLANTATION (2021)

Article Hematology

Improved stenosis outcome in stroke-free sickle cell anemia children after transplantation compared to chronic transfusion

Suzanne Verlhac et al.

Summary: In stroke-free children with sickle cell anaemia, stem cell transplantation significantly improved stenosis outcomes over 3 years compared to standard-care, indicating better prevention and improved outcome.

BRITISH JOURNAL OF HAEMATOLOGY (2021)

Article Hematology

Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres

Mohsen Alzahrani et al.

Summary: Non-myeloablative HPCT from MRD has shown to be effective and well-tolerated in treating SCD patients, with high survival rates and low incidence of clinically significant GVHD. Organ functions improved post-transplantation and further sickle-related injuries were halted, suggesting the success of the treatment regimen.

BRITISH JOURNAL OF HAEMATOLOGY (2021)

Article Oncology

Is Busulfan Clearance Different in Patients With Sickle Cell Disease? Let's Clear Up That Case With Some Controls

Amandine Remy et al.

Summary: This study found that SCD does not significantly affect the metabolic clearance rate of the first dose of busulfan, regardless of genetic or anthropometric factors considered.

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY (2021)

Article Biophysics

Successful mismatched hematopoietic stem cell transplantation for pediatric hemoglobinopathy by using ATG and post-transplant cyclophosphamide

Lisa V. E. Oostenbrink et al.

Summary: The use of mismatched unrelated or haploidentical donors in combination with ATG and PT-CY for pediatric patients with hemoglobinopathies can be considered a safe and effective treatment option when an HLA-identical or matched unrelated donor is not available.

BONE MARROW TRANSPLANTATION (2021)

Article Economics

Indirect Economic Burden of Sickle Cell Disease

David Holdford et al.

Summary: This study aimed to quantify the indirect costs of sickle cell disease in the United States and found that pain events had a significant negative impact on employment status, productivity, and daily lives of adults with the disorder.

VALUE IN HEALTH (2021)

Article Medicine, Research & Experimental

Antibody-drug conjugates plus Janus kinase inhibitors enable MHC-mismatched allogeneic hematopoietic stem cell transplantation

Stephen P. Persaud et al.

Summary: The study demonstrates that using ADC conditioning in mice can achieve robust multilineage allogeneic engraftment. Mechanistic studies of JAK1/2 inhibition revealed impairments in T and NK cell survival, proliferation, and effector function. The combination of ADCs and baricitinib balanced graft-versus-host disease and graft-versus-leukemia responses.

JOURNAL OF CLINICAL INVESTIGATION (2021)

Article Hematology

Fertility Concerns and Access to Care for Stem Cell Transplantation Candidates with Sickle Cell Disease

Adrienne D. Mishkin et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2020)

Review Medicine, General & Internal

Patient-Reported Outcomes and Economic Burden of Adults with Sickle Cell Disease in the United States: A Systematic Review

Soyon Lee et al.

INTERNATIONAL JOURNAL OF GENERAL MEDICINE (2020)

Article Emergency Medicine

Trends in Sickle Cell Disease-Related Mortality in the United States, 1979 to 2017

Amanda B. Payne et al.

ANNALS OF EMERGENCY MEDICINE (2020)

Article Hematology

Nonmyeloablative Matched Sibling Donor Hematopoietic Cell Transplantation in Children and Adolescents with Sickle Cell Disease

Gregory M. T. Guilcher et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2019)

Article Biophysics

Haploidentical CD3 or α/β T-cell depleted HSCT in advanced stage sickle cell disease

Juergen Foell et al.

BONE MARROW TRANSPLANTATION (2019)

Review Allergy

Busulfan-Induced Lung Injury in Pediatric Oncology Patients-Review of the Literature with an Illustrative Case

Nusa Matijasic et al.

PEDIATRIC ALLERGY IMMUNOLOGY AND PULMONOLOGY (2019)

Article Hematology

HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease

Anna B. Pawlowska et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2018)

Article Biotechnology & Applied Microbiology

Myeloid Conditioning with c-kit-Targeted CAR-T Cells Enables Donor Stem Cell Engraftment

Yasuyuki Arai et al.

MOLECULAR THERAPY (2018)

Article Obstetrics & Gynecology

Risks associated with fertility preservation for women with sickle cell anemia

Lydia H. Pecker et al.

FERTILITY AND STERILITY (2018)

Article Medicine, General & Internal

Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease

K. I. Ataga et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Hematology

Alternative donor allogeneic hematopoietic cell transplantation for hemoglobinopathies

Feras Alfraih et al.

SEMINARS IN HEMATOLOGY (2016)

Article Medicine, General & Internal

Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype

Matthew M. Hsieh et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2014)

Article Medicine, Research & Experimental

Induction of Tolerance through Mixed Chimerism

David H. Sachs et al.

COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2014)

Article Hematology

Stem cell transplantation after reduced-intensity conditioning for sickle cell disease

Susanne Matthes-Martin et al.

EUROPEAN JOURNAL OF HAEMATOLOGY (2013)

Article Obstetrics & Gynecology

Toxicity of Chemotherapy and Radiation on Female Reproduction

Dror Meirow et al.

CLINICAL OBSTETRICS AND GYNECOLOGY (2010)

Article Hematology

Defining the Intensity of Conditioning Regimens: Working Definitions

Andrea Bacigalupo et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2009)

Article Hematology

Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease

Lakshmanan Krishnamurti et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2008)

Article Oncology

Children with sickle cell disease: Growth and gonadal function after hematopoietic stem cell transplantation

Cecile Brachet et al.

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY (2007)

Article Hematology

Myeloablative and immunosuppressive properties of treosulfan in mice

F Sjoo et al.

EXPERIMENTAL HEMATOLOGY (2006)

Article Hematology

Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia

MC Walters et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2001)