Jerry Stein
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View article: A Nationwide Analysis of the Phenotype/Genotype Landscape of Hemophagocytic Lymphohistiocytosis: UNC13D Associates with Poor Prognosis
A Nationwide Analysis of the Phenotype/Genotype Landscape of Hemophagocytic Lymphohistiocytosis: UNC13D Associates with Poor Prognosis Open
Background/objectives: Geographic and ethnic differences influence the genetic landscape of hemophagocytic lymphohistiocytosis (HLH) and the frequency of familial HLH (FHL); this in turn can affect outcomes. Methods: We collected data on 9…
View article: Granulocyte Transfusions in Pediatric Hemato‐Oncology: Neutrophil Response and CRP Kinetics
Granulocyte Transfusions in Pediatric Hemato‐Oncology: Neutrophil Response and CRP Kinetics Open
Background Granulocyte transfusions (GTXs) are used in neutropenic pediatric hemato‐oncology patients with infections unresponsive to antimicrobial therapy, but their clinical benefit remains uncertain. Methods We retrospectively reviewed …
View article: Update on long-term outcomes of a cohort of patients with TCF3::HLF positive acute lymphoblastic leukemia treated with blinatumomab and stem cell transplantation
Update on long-term outcomes of a cohort of patients with TCF3::HLF positive acute lymphoblastic leukemia treated with blinatumomab and stem cell transplantation Open
Not available.
View article: Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM
Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM Open
The superiority of total body irradiation (TBI)–based vs chemotherapy conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with acute lymphoblastic leukemia (ALL) has been established in the internati…
View article: Plasma exchange using peripheral arterial and venous access in the pediatric intensive care unit
Plasma exchange using peripheral arterial and venous access in the pediatric intensive care unit Open
Objective Therapeutic plasma exchange (TPE) is a vital therapeutic modality in pediatric intensive care units (PICU) for various indications. Traditionally, pediatric TPE is performed via a large bore, double lumen catheter, whose insertio…
View article: Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the <scp>I‐BFM</scp> Study Group
Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the <span>I‐BFM</span> Study Group Open
Summary This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan‐based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic my…
View article: Comparable Outcome after Busulfan- or Treosulfan-Based Conditioning Regimen in Children Above 4 Years of Age with ALL Undergoing Allogeneic HSCT. Results from the Prospective International Forum-Trial
Comparable Outcome after Busulfan- or Treosulfan-Based Conditioning Regimen in Children Above 4 Years of Age with ALL Undergoing Allogeneic HSCT. Results from the Prospective International Forum-Trial Open
Purpose: Total body irradiation (TBI) has proved to be the “gold standard” as part of the conditioning regimen before allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL). …
View article: Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study
Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study Open
Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late …
View article: Combined Kidney and Hematopoietic Cell Transplantation for Tolerance Induction between HLA Matched Sibling Donor-Recipient Pairs
Combined Kidney and Hematopoietic Cell Transplantation for Tolerance Induction between HLA Matched Sibling Donor-Recipient Pairs Open
A variety of options exist to reconstruct limbs following traumatic bone loss. The management of these injuries is challenging and often requires prolonged and potentially painful treatment. The Ilizarov technique of bone transport using c…
View article: DPP9 deficiency: An inflammasomopathy that can be rescued by lowering NLRP1/IL-1 signaling
DPP9 deficiency: An inflammasomopathy that can be rescued by lowering NLRP1/IL-1 signaling Open
Dipeptidyl peptidase 9 (DPP9) is a direct inhibitor of NLRP1, but how it affects inflammasome regulation in vivo is not yet established. Here, we report three families with immune-associated defects, poor growth, pancytopenia, and skin pig…
View article: Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation: Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)
Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation: Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) Open
Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international pr…
View article: Total Body Irradiation or Chemotherapy Conditioning in Childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study
Total Body Irradiation or Chemotherapy Conditioning in Childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study Open
PURPOSE Total body irradiation (TBI) before allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL) is efficacious, but long-term side effects are concerning. We investigated …
View article: Outcome of children relapsing after first allogeneic haematopoietic stem cell transplantation for acute myeloid leukaemia: a retrospective I‐BFM analysis of 333 children
Outcome of children relapsing after first allogeneic haematopoietic stem cell transplantation for acute myeloid leukaemia: a retrospective I‐BFM analysis of 333 children Open
Summary Outcome of 333 children with acute myeloid leukaemia relapsing after a first allogeneic haematopoietic stem cell transplantation was analyzed. Four‐year probability of overall survival (4y‐pOS) was 14%. 4y‐pOS for 122 children rece…
View article: Children and Adults with Refractory Acute Graft-versus-Host Disease Respond to Treatment with the Mesenchymal Stromal Cell Preparation “MSC-FFM”—Outcome Report of 92 Patients
Children and Adults with Refractory Acute Graft-versus-Host Disease Respond to Treatment with the Mesenchymal Stromal Cell Preparation “MSC-FFM”—Outcome Report of 92 Patients Open
(1) Background: Refractory acute graft-versus-host disease (R-aGvHD) remains a leading cause of death after allogeneic stem cell transplantation. Survival rates of 15% after four years are currently achieved; deaths are only in part due to…
View article: Durable remissions in <i>TCF3-HLF</i> positive acute lymphoblastic leukemia with blinatumomab and stem cell transplantation
Durable remissions in <i>TCF3-HLF</i> positive acute lymphoblastic leukemia with blinatumomab and stem cell transplantation Open
Persisting clones in compartments other than bone marrow may not be covered by MRD quantification but could still be responsive to blinatumomab therapy.The responses detected in this TCF3-HLF-positive ALL cohort are encouraging and suggest…
View article: Treosulfan induces distinctive gonadal toxicity compared with busulfan
Treosulfan induces distinctive gonadal toxicity compared with busulfan Open
Treosulfan (L-treitol-1,4-bis-methanesulfonate) has been increasingly incorporated as a main conditioning protocol for hematopoietic stem cell transplantation in pediatric malignant and non-malignant diseases. Treosulfan presents lower tox…
View article: Apograft, a Novel Stem Cell Selection Technology, Prevents Graft vs. Host Disease (GvHD) While Preserving Graft vs Leukemia (GvL) Effects
Apograft, a Novel Stem Cell Selection Technology, Prevents Graft vs. Host Disease (GvHD) While Preserving Graft vs Leukemia (GvL) Effects Open
Background: Despite pharmacological prophylaxis and treatment, GvHD remains a common and often severe complication of allogeneic hematopoietic stem cell transplantation (HSCT). Complete donor T cell depletion from the graft prevents GvHD, …
View article: Disruption of Thrombocyte and T Lymphocyte Development by a Mutation in <i>ARPC1B</i>
Disruption of Thrombocyte and T Lymphocyte Development by a Mutation in <i>ARPC1B</i> Open
Regulation of the actin cytoskeleton is crucial for normal development and function of the immune system, as evidenced by the severe immune abnormalities exhibited by patients bearing inactivating mutations in the Wiskott–Aldrich syndrome …
View article: Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin
Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin Open
Key Points Germ line biallelic loss-of-function THPO mutations cause BMF. Marrow failure due to THPO mutations is characterized by poor graft function after transplantation but responds to THPO receptor agonists.
View article: East Meets West—Impact of Ethnicity on Donor Match Rates in the Ezer Mizion Bone Marrow Donor Registry
East Meets West—Impact of Ethnicity on Donor Match Rates in the Ezer Mizion Bone Marrow Donor Registry Open
HLA haplotype frequencies in a volunteer bone marrow donor registry should reflect the frequencies of potential transplant recipients served by that registry, a challenge in a country with diverse subethnicities of immigrants from Eastern …
View article: Optimizing the process of fertility preservation in pediatric female cancer patients – a multidisciplinary program
Optimizing the process of fertility preservation in pediatric female cancer patients – a multidisciplinary program Open
Multidisciplinary programs that encompass experts of all relevant fields, skilled laboratory resources and a facilitated path appear to maximize the yield. We observed a considerable higher referral rates following launching a designated p…
View article: Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis
Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis Open
Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for most children with osteopetrosis (OP). Timing of HSCT is critical; therefore, umbilical cord blood transplantation (UCBT) is an attractive option. We analyze…