Patrick Lutz
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View article: Astrocytes and pericytes attenuate severely injured patient plasma mediated expression of tight junction proteins in endothelial cells
Astrocytes and pericytes attenuate severely injured patient plasma mediated expression of tight junction proteins in endothelial cells Open
Blood Brain Barrier (BBB) breakdown is a secondary form of brain injury which has yet to be fully elucidated mechanistically. Existing research suggests that breakdown of tight junction proteins between endothelial cells is a primary drive…
View article: Genotype/phenotype correlations of childhood‐onset congenital sideroblastic anaemia in a European cohort
Genotype/phenotype correlations of childhood‐onset congenital sideroblastic anaemia in a European cohort Open
Summary Congenital sideroblastic anaemia (CSA) is a rare disease caused by germline mutations of genes involved in haem and iron‐sulphur cluster formation, and mitochondrial protein biosynthesis. We performed a retrospective multicentre Eu…
View article: Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France
Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France Open
Allogeneic stem cell transplantation remains the only curative treatment for sickle cell anemia (SCA), but the place of myeloablative conditioning in the procedure remains to be defined. The aim of the present study was to analyze long-ter…
View article: Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease
Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease Open
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative therapy for sickle cell disease (SCD).41 Several barriers prevent its widespread application, including the lack of a suitable donor, risk of early and lat…
View article: Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience
Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience Open
In this retrospective study, we evaluate long-term complications in nearly all β-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a m…
View article: Prolonged <i>versus</i> standard native <i>E. coli</i> asparaginase therapy in childhood acute lymphoblastic leukemia and non-Hodgkin lymphoma: final results of the EORTC-CLG randomized phase III trial 58951
Prolonged <i>versus</i> standard native <i>E. coli</i> asparaginase therapy in childhood acute lymphoblastic leukemia and non-Hodgkin lymphoma: final results of the EORTC-CLG randomized phase III trial 58951 Open
Asparaginase is an essential component of combination chemotherapy for childhood acute lymphoblastic leukemia and non-Hodgkin lymphoma. The value of asparaginase was further addressed in a group of non-very high-risk patients by comparing …
View article: Benefits of rituximab as a second‐line treatment for autoimmune haemolytic anaemia in children: a prospective French cohort study
Benefits of rituximab as a second‐line treatment for autoimmune haemolytic anaemia in children: a prospective French cohort study Open
Summary Childhood autoimmune haemolytic anaemia ( AIHA ) requires second‐line immunosuppressive therapy in 30–50% of cases. It appears that rituximab is indicated in such circumstances. This prospective national study reports the practice,…
View article: Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation Open
Key Points HLA-identical sibling transplantation for SCD offers excellent long-term survival. Mortality risk is higher for older patients; event-free survival has improved in patients transplanted after 2006.
View article: Cutaneous malignant melanoma in children and adolescents treated in pediatric oncology units
Cutaneous malignant melanoma in children and adolescents treated in pediatric oncology units Open
Objectives Recent progress in the understanding of tumor biology and new targeted therapies has led to improved survival in adults with malignant melanoma (MM). MM is rare in children, especially before puberty. We report here our experien…
View article: Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome
Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome Open
Key Points A double-unit strategy does not decrease transplantation failure risk when a single unit of cord blood with adequate cell dose is available. Alloreactivity may be enhanced by double-unit cord blood transplantation.
View article: Impact of therapy in a cohort of unselected children with Down Syndrome‐associated Acute Lymphoblastic Leukaemia
Impact of therapy in a cohort of unselected children with Down Syndrome‐associated Acute Lymphoblastic Leukaemia Open
Down syndrome (DS) children account for approximately 3% of children with acute lymphoblastic leukaemia (ALL) and 5–8% of children with acute myeloid leukaemia (AML) (Maloney et al, 2010). Most reports indicate that DS-ALL patients fare wo…
View article: Evans Syndrome in Children: Long-Term Outcome in a Prospective French National Observational Cohort
Evans Syndrome in Children: Long-Term Outcome in a Prospective French National Observational Cohort Open
Evans syndrome (ES) is a rare autoimmune disorder whose long-term outcome is not well known. In France, a collaborative pediatric network set up via the National Rare Disease Plan now provides comprehensive clinical data in children with t…
View article: Response: Confounding by indication is unlikely to explain the higher inhibitor incidence in boys treated with a recombinant FVIII product
Response: Confounding by indication is unlikely to explain the higher inhibitor incidence in boys treated with a recombinant FVIII product Open
In January 2013, the Research of Determinants of Inhibitor Development (RODIN) study unexpectedly showed a higher inhibitor incidence in previously untreated patients (PUPs) with severe hemophilia A treated with Kogenate FS (Bayer) (also n…