Results 151 to 160 of about 276,679 (267)

Clinical Predictors of Response in Chronic Graft‐Versus‐Host Disease: Results From the “Predicting the Quality of Response to Specific Treatments (PQRST)” Trial

American Journal of Hematology, EarlyView.
ABSTRACT Despite significant progress in chronic GVHD therapies, challenges remain in understanding pleomorphic phenotypes and varying responses to treatment. The aim of this study was to identify predictors of treatment response. We conducted a prospective, observational cohort study of patients beginning first‐, second‐, or third‐line systemic ...
Betty K. Hamilton, Lynn Onstad, Paul A. Carpenter, Joseph Pidala, George Chen, Corey Cutler, Najla El Jurdi, Nosha Farhadfar, Mark Juckett, Carrie L. Kitko, Catherine J. Lee, Stephanie J. Lee   +11 more
wiley   +1 more source

Allogeneic hematopoietic stem cell transplantation in ERCC6L2 disease. [PDF]

Blood Adv
Hakkarainen M, Sicre de Fontbrune F, Kaaja I, Douglas S, Dalle JH, Risitano AM, Kulasekararaj A, Alsultan A, Cutler C, Ho VT, Hellström-Lindberg E, Mielke S, Myhre AE, Rihani R, Shanap MA, Hashem H, Shimamura A, Rowe RG, Auer F, Beier F, Desnica L, Hough R, Ali Jafri SR, Ayas M, Jardine L, Mellid EF, Corrales I, Richardson D, Özbek NY, Zaniewska-Tekieli A, Gozdzik J, Ryhänen S, Niinimäki R, Jahnukainen K, Salmenniemi U, Kilpivaara O, Wartiovaara-Kautto U.   +36 more
europepmc   +1 more source

Dordaviprone Maintenance After Allogeneic HCT for High‐Risk Acute Myeloid Leukemia and Myelodysplastic Neoplasm

American Journal of Hematology, EarlyView.
ABSTRACT Dordaviprone is a first‐in‐class small‐molecule imipridone. In preclinical studies, it is active against leukemia cells harboring a TP53 mutation or complex karyotype and leukemia stem cells while sparing normal bone marrow cells. This study aimed to determine the safety of dordaviprone maintenance after allogeneic hematopoietic cell ...
Vijaya Raj Bhatt, Christopher S. Wichman, Alyssa Bouska, Moataz Ellithi, Michael Haddadin, Javeed Iqbal, James E. Talmadge, Lori J. Maness, Krishna Gundabolu   +8 more
wiley   +1 more source

Hematopoietic Stem-Cell Gene Therapy for Cystinosis. [PDF]

N Engl J Med
Barshop BA, Ball ED, Benador N, Trauner D, Phillips S, Dohil R, Afshari NA, Roy S, Campo Fernandes B, Kohn D, Shayan K, Everett JK, Bushman FD, Midgley J, Liang H, Sawyers A, Gangoiti JA, Panchal M, Ahmed I, Cherqui S.   +19 more
europepmc   +1 more source

Undetectable Hydroxyurea Levels in the Majority of Sickle Cell Disease Patients, Especially in Young Children

American Journal of Hematology, EarlyView.
ABSTRACT Hydroxyurea (HU) is the most widely prescribed disease‐modifying treatment in sickle cell disease (SCD), though treatment responses vary due to metabolism and adherence. We examined HU blood levels and treatment response in patients with homozygous sickle cell disease (HbSS).
Sigrid van der Veen, Bart J. Biemond, Marjon H. Cnossen, Pablo Bartolucci, Maria P. Boaro, Betzabel Cajiao Garcia, Raffaella Colombatti, M. D'Agnolo, Karin Fijnvandraat, Anna Collado Gimbert, Amira Idrizovic, Petros Kountouris, Mar Mañú‐Pereira, Elisabetta Mezzalira, Mariane de Montalembert, Erfan Nur, A. Pham Hung D' Alexandry D'Orengiani, Tiziana Sanavia, Saskia E. M. Schols, Marissa J. M. Traets, Minke A. E. Rab, Sara I. Reidel, Anita W. Rijneveld, Yosr Zaouali, Nanda M. Verhoeven‐Duif, Judith J. M. Jans, Eduard J. van Beers   +26 more
wiley   +1 more source

PB2460: THE RATE OF NEUTROPHIL RECOVERY IS HELPFUL IN PREDICTING ACUTE GRAFT-VERSUS-HOST DISEASE AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION: AN OBSERVATIONAL COHORT STUDY

HemaSphere, 2023
Sihan Lai, Xue LI, Ying He, Shan Zhang, Guangcui He, Yan Deng, Ying Han, Hai Yi   +7 more
doaj   +1 more source

High Proportion of PNH Type II Neutrophils Is Associated With Thrombosis in Patients Displaying a PNH Clone ≥ 1%

American Journal of Hematology, EarlyView.
ABSTRACT The clinical significance of PNH Type II white blood cells (WBCs) remains unclear. We assessed the relative percentage (rel%) of Type II neutrophils in 355 patients with a PNH clone ≥ 1% on neutrophils enrolled by 33 flow cytometry laboratories in the 5‐year French nation‐wide multicenter prospective observational study.
Orianne Wagner‐Ballon, Anne‐Catherine Lhoumeau, Anna Raimbault, Camille Debord, Jean‐Philippe Vial, Valérie Soenen, Mélanie Pannetier, Edouard Bonneville, Elsa Bera, Caroline Bret, Nicolas Chapuis, Véronique Latger‐Cannard, Caroline Mayeur‐Rousse, Véronique Harrivel, François Vergez, Marie Loosveld, Juliette Vo Xuan, Stéphanie Mathis, Gérard Socié, Flore Sicre de Fontbrune, Régis Peffault de Latour, Bernard Drenou, Agathe Debliquis, Magali Le Garff‐Tavernier, on behalf of the French PNH working group CytHem‐HPN, Thomas Boyer, Mikaël Roussel, Marie Guillon, Sabrina Bouyer, Isabelle Kittler, Franck Geneviève, Julien Guy, Sophie Brun, Claude Capron, Sébastien Lachot, Francine Garnache‐Ottou, Richard Veyrat‐Masson, Corine Brunet, Caren Brumpt, Ludovic Lhermitte. Finally, Louis Terriou, Nicolas Blin   +41 more
wiley   +1 more source

Telomere length and clonal hematopoiesis interact to influence outcomes in hematopoietic stem cell transplantation. [PDF]

Blood Adv
Tobin J, Stelmach P, Richter S, Xu D, Sauer S, Raab MS, Gu M, Dunn WG, Fabre MA, Müller-Tidow C, Vassiliou GS.   +10 more
europepmc   +1 more source

The Critical Role of Fractionated Urine Glycosaminoglycans in the Evaluation of Mucopolysaccharidosis Type II in Four Unrelated Families

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Since 2015, Ann and Robert H. Lurie Children's Hospital has performed diagnostic testing for infants who screen positive for mucopolysaccharidosis type II (MPS II) on the Illinois newborn screen. Preliminary diagnostic testing includes measurement of plasma iduronate‐2‐sulfatase enzyme activity and urinary glycosaminoglycan analysis, followed ...
Carly A. Rasmussen, Monika Izdebski, Allison Paltzer, Rachel Hickey, Laura Pollard, Joshua Baker   +5 more
wiley   +1 more source

Hematopoietic Stem Cell Transplant and Brain Volume Changes in Adults With Sickle Cell Disease. [PDF]

Neurology
Kern KC, Inam Z, Hsieh MM, Tisdale JF, Fitzhugh CD, Limerick EM, Gebremeskel ASK, White T, Jordan LC, Lynch JK.   +9 more
europepmc   +1 more source