David C. Shyr
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View article: Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report
Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report Open
Wiskott-Aldrich syndrome (WAS), an X-linked disorder characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancy, can be effectively treated with allogeneic hematopoietic cell transplantation (HCT). Older age at HCT a…
View article: Exploring Pattern of Relapse in Pediatric Patients with Acute Lymphocytic Leukemia and Acute Myeloid Leukemia Undergoing Stem Cell Transplant Using Machine Learning Methods
Exploring Pattern of Relapse in Pediatric Patients with Acute Lymphocytic Leukemia and Acute Myeloid Leukemia Undergoing Stem Cell Transplant Using Machine Learning Methods Open
Background. Leukemic relapse remains the primary cause of treatment failure and death after allogeneic hematopoietic stem cell transplant. Changes in post-transplant donor chimerism have been identified as a predictor of relapse. A better …
View article: Exploring Pattern of Relapse in Pediatric Patients with Acute Lymphocytic Leukemia and Acute Myeloid Leukemia Undergoing Stem Cell Transplant Using Machine Learning Methods
Exploring Pattern of Relapse in Pediatric Patients with Acute Lymphocytic Leukemia and Acute Myeloid Leukemia Undergoing Stem Cell Transplant Using Machine Learning Methods Open
In this report, we present a study demonstrating how machine learning methods can reveal the interaction of different risk factors of post-transplant leukemic relapse and obtain robust predictions even with a modest clinical dataset. Using…
View article: Stem cell transplantation for ALL: you've always got a donor, why not always use it?
Stem cell transplantation for ALL: you've always got a donor, why not always use it? Open
Hematopoietic stem cell transplantation (HSCT) represents a consolidated therapeutic strategy for high-risk pediatric acute lymphoblastic leukemia (ALL), offering the potential for curative treatment. This manuscript delves into the debate…
View article: Longitudinal clinical data improve survival prediction after hematopoietic cell transplantation using machine learning
Longitudinal clinical data improve survival prediction after hematopoietic cell transplantation using machine learning Open
Serial prognostic evaluation after allogeneic hematopoietic cell transplantation (allo-HCT) might help identify patients at high risk of lethal organ dysfunction. Current prediction algorithms based on models that do not incorporate change…
View article: Precision Delivery of Steroids as a Rescue Therapy for Gastrointestinal Graft-versus-Host Disease in Pediatric Stem Cell Transplant Recipients
Precision Delivery of Steroids as a Rescue Therapy for Gastrointestinal Graft-versus-Host Disease in Pediatric Stem Cell Transplant Recipients Open
Graft versus host disease (GVHD) is one of the most serious complications following stem cell transplant in children and is a major cause of morbidity and mortality. Corticosteroids remain the mainstay of treatment, and although a majority…
View article: Effect of Testicular Boost in Children With Leukemia Receiving Total Body Irradiation and Stem Cell Transplant: A Single-Institution Experience
Effect of Testicular Boost in Children With Leukemia Receiving Total Body Irradiation and Stem Cell Transplant: A Single-Institution Experience Open
Omission of testicular boost may be associated with comparable oncologic but improved gonadal endocrine outcomes and should be further studied.
View article: Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC Open
Adenosine deaminase (ADA) deficiency causes ∼13% of cases of severe combined immune deficiency (SCID). Treatments include enzyme replacement therapy (ERT), hematopoietic cell transplant (HCT), and gene therapy (GT). We evaluated 131 patien…
View article: Predicting Relapse in Pediatric Patients with Acute Lymphoblastic Leukemia Undergoing Stem Cell Transplant Using Interpretable Machine Learning—Applying Machine Learning Beyond 'Big Data'
Predicting Relapse in Pediatric Patients with Acute Lymphoblastic Leukemia Undergoing Stem Cell Transplant Using Interpretable Machine Learning—Applying Machine Learning Beyond 'Big Data' Open
View article: Ruxolitinib As a Salvage Therapy for Acute and Chronic Graft-versus-Host Disease in Children and Young Adults: a single institution experience
Ruxolitinib As a Salvage Therapy for Acute and Chronic Graft-versus-Host Disease in Children and Young Adults: a single institution experience Open
Background Ruxolitinib, a Janus kinase (JAK)-1/JAK-2 inhibitor that reduces T-cell activation and proliferation leading to a potent anti-inflammatory effect, was recently approved to treat steroid-refractory acute graft-versus-host disease…
View article: Impact of Testicular Boost on Oncologic Outcomes and Late Effects in Pediatric Patients With Leukemia Receiving Fractionated Total Body Irradiation (TBI): A Single-Institution Experience
Impact of Testicular Boost on Oncologic Outcomes and Late Effects in Pediatric Patients With Leukemia Receiving Fractionated Total Body Irradiation (TBI): A Single-Institution Experience Open
View article: Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers
Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers Open
View article: Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers
Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers Open
View article: Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report Open
Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the prima…
View article: Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey
Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey Open
Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated wi…
View article: Transplantation Outcomes for Children with Severe Combined Immune Deficiency (SCID) Have Improved over Time: A 36-Year Summary Report By the Primary Immune Deficiency Treatment Consortium (PIDTC)
Transplantation Outcomes for Children with Severe Combined Immune Deficiency (SCID) Have Improved over Time: A 36-Year Summary Report By the Primary Immune Deficiency Treatment Consortium (PIDTC) Open
With >36 years of data collected, PIDTC prospective (6901) and retrospective (6902) natural history studies provide an unprecedented opportunity to study hematopoietic cell transplantation (HCT) outcomes for SCID over time. Patients in thi…
View article: Machine Learning Applications in the Diagnosis of Benign and Malignant Hematological Diseases
Machine Learning Applications in the Diagnosis of Benign and Malignant Hematological Diseases Open
The use of machine learning (ML) and deep learning (DL) methods in hematology includes diagnostic, prognostic, and therapeutic applications. This increase is due to the improved access to ML and DL tools and the expansion of medical data. …
View article: Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network
Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network Open
The leukodystrophies are a heterogeneous group of inherited diseases characterized by progressive demyelination of the central nervous system leading to devastating neurologic symptoms and premature death. Hematopoietic stem cell transplan…
View article: Inflammatory Bowel Disease in Patients with Chronic Granulomatous Disease May Be Under-Recognized
Inflammatory Bowel Disease in Patients with Chronic Granulomatous Disease May Be Under-Recognized Open
View article: The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018)
The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018) Open
View article: SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery
SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery Open
Key Points The genetic cause of SCID impacts on survival and immune reconstitution and should be considered in tailoring HCT for individual patients. Total and naive CD4+ cell counts in SCID patients 6 and 12 months post-HCT predict long-t…
View article: B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation
B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation Open
Key Points IL2RG/JAK3-deficient B cells remain intrinsically defective posttransplant despite follicular helper T-cell reconstitution. In vitro response of B cells to IL-21 is a potential biomarker for humoral immunity in patients with IL2…
View article: Single Center Experience with Total Nucleated Cell, CD34 and CD3 Cell Yield From Bone Marrow Harvests in Both Adult and Pediatric Donors
Single Center Experience with Total Nucleated Cell, CD34 and CD3 Cell Yield From Bone Marrow Harvests in Both Adult and Pediatric Donors Open
With the popularization of T-cell replete haploidentical stem cell transplant, bone marrow grafts have become more utilized due to lower risk of GVHD and comparable overall survival to peripheral blood stem cell grafts [1Bashey A. Zhang M.…
View article: Genotype, Phenotype and T Cell Counts at One Year Predict Survival and Long Term Immune Reconstitution after Transplantation in Severe Combined Immune Deficiency (SCID)—The Primary Immune Deficiency Treatment Consortium (PIDTC)
Genotype, Phenotype and T Cell Counts at One Year Predict Survival and Long Term Immune Reconstitution after Transplantation in Severe Combined Immune Deficiency (SCID)—The Primary Immune Deficiency Treatment Consortium (PIDTC) Open
Patients: We retrospectively studied 581 patients with typical SCID who received a hematopoietic cell transplant (HCT) at 32 PIDTC centers from 1982-2012. Known mutations (n = 355) included 178 gC, 35 JAK3, 40 IL7R, 6 CD3, 1 CD45, 27 DCLRE…