Turnpenny-Fry Syndrome (TPFS) is a rare genetic disorder characterized by a severe developmental delay and a distinctive facial gestalt. It is caused by mutations in the Polycomb Group Ring Finger Protein 2 (PCGF2) gene, which is also known to play a role in numerous tumor types. Up to date, there have been no published case reports of patients with TPFS and concomitant malignancies. The present case describes the clinical evaluation and follow-up of a male infant with severe global developmental delay (GDD) and a distinctive phenotype. At 4 years of age, clinical exome sequencing confirmed the diagnosis of TPFS. Posteriorly, at 5 years of age, the patient was also diagnosed with T-cell acute lymphoblastic leukemia (ALL). Given the scarce literature regarding this syndrome, the authors expect that this case report will provide valuable information that could improve the follow-up of patients with TPFS. Furthermore, this case highlights the necessity for the appropriate diagnosis of developmental disorders, to ensure adequate care, surveillance of comorbidities and proper genetic counselling.
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http://dx.doi.org/10.7759/cureus.53099 | DOI Listing |
Leukemia
January 2025
Australian Centre for Blood Diseases (ACBD), School of Translational Medicine, Monash University, Melbourne, VIC, Australia.
Early T-cell Precursor Acute Lymphoblastic Leukemia (ETP-ALL) is an immature subtype of T-cell acute lymphoblastic leukemia (T-ALL) commonly show deregulation of the LMO2-LYL1 stem cell transcription factors, activating mutations of cytokine receptor signaling, and poor early response to intensive chemotherapy. Previously, studies of the Lmo2 transgenic mouse model of ETP-ALL identified a population of stem-like T-cell progenitors with long-term self-renewal capacity and intrinsic chemotherapy resistance linked to cellular quiescence. Here, analyses of Lmo2 transgenic mice, patient-derived xenografts, and single-cell RNA-sequencing data from primary ETP-ALL identified a rare subpopulation of leukemic stem cells expressing high levels of the cytokine receptor FLT3.
View Article and Find Full Text PDFJCO Glob Oncol
January 2025
Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada.
Purpose: Patients with adolescent and young adult (AYA) cancer are recognized as a vulnerable subpopulation in high-income countries (HICs). Although survival gaps between HIC and low- and middle-income country (LMIC) children with cancer are well described, LMIC AYAs have been neglected. We conducted a systematic review to describe cancer outcomes among LMIC AYAs.
View Article and Find Full Text PDFInt J Hematol
January 2025
Department of Hematology, The 920th Hospital of Joint Logistics Support Force, No.212, Da Guan Road, Xishan District, Kunming, 650100, Yunnan, China.
Background: The treatment of relapsed/refractory T cell acute lymphoblastic leukemia (R/R T-ALL) is a significant challenge in hematologic oncology, and no standard salvage treatment plan exists. Both Chinese and international clinical guidelines recommend combination chemotherapy including venetoclax.
Methods: Efficacy and safety of venetoclax, azacitidine, homoharringtonine, cytarabine, and aclarubicin (VA-HAA) combination therapy were retrospectively analyzed in 3 patients with R/R T-ALL at the Department of Hematology, 920th Hospital of the Joint Logistics Support Force, Chinese People's Liberation Army.
Ann Hematol
January 2025
Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.
Early T-precursor acute lymphoblastic leukemia/lymphoma (ETP-ALL/LBL) is a rare and aggressive subtype of T-cell leukemia with poor prognosis and resistance to standard treatments. We report a 21-year-old male with ETP-ALL/LBL who, after an initial complete remission with the HOELZER protocol, experienced early relapse and was refractory to subsequent FLEND and BFM protocols. Following disease progression and complications, he was treated with a combination of daratumumab, venetoclax, azacitidine, and dexamethasone.
View Article and Find Full Text PDFActa Endocrinol (Buchar)
January 2025
Dokuz Eylül University, Faculty of Medicine, Division of Pediatric Endocrinology.
Context: Osteonecrosis (ON) is bone death caused by inadequate blood supply and its optimal management remains uncertain.
Objective: We describe the outcomes of BP (pamidronate) treatment in our patients.
Design: Data regarding clinical, laboratory, magnetic resonance imaging (MRI) studies, and bone mineral density measurements (BMD) were recorded before and one year after treatment (reevaluation).
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