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Pediatric Oncology
Published in Pat Price, Karol Sikora, Treatment of Cancer, 2020
Stephen Lowis, Rachel Cox, John Moppett, Helen Rees
The pivotal study for pediatric ALL is the Eliana study of CTL-019, Tisagenlecleucel.12 This study of 75 patients with relapsed refractory B-cell precursor ALL showed a 50% 12-month EFS in a group of patients with a median of three prior therapies. Importantly, there was no relationship between efficacy and the number of prior therapies including SCT. A common reason for failure was CD19-negative relapse. It should be noted that in all successful cases to date, prolonged B-cell aplasia has been a long-term side effect, though this has been effectively managed with immunoglobulin replacement therapy.
Dyskeratosis Congenita
Published in Dongyou Liu, Handbook of Tumor Syndromes, 2020
The BM findings in DC are variable and range from normal to different severity of aplasia depending on the stage of the disease. Aplastic anemia occurs in patients with a median age of presentation of 11 years. The anemia is macrocytic. Fetal hemoglobin levels are increased. Approximately 90% have peripheral cytopenia of one or more lineages. Initially, the patient usually presents thrombocytopenia, and during the evolution of the disease this becomes more global and they develop severe BMF. BMF (in up to 90% of patients) is one of the major causes of premature mortality in DC due to opportunistic infections because of the significant reduction in mature blood cells. The BM abnormalities can progress in different forms with the appearance of myelodysplasia in one or more lineages or leukemia. It has been reported that patients with DC have an observed expected ratio of 2663 to develop myelodysplasia (95% confidence interval 858–6215), with a mean age of onset of 35 years.
Genetic Causes of Male Infertility
Published in Botros Rizk, Ashok Agarwal, Edmund S. Sabanegh, Male Infertility in Reproductive Medicine, 2019
Amr Abdel Raheem, Mohamed Wael Ragab, Tarek M. A. Aly
Diagnosis of vasal aplasia is mainly made by clinical examination. The vas is a firm tube in the posteromedial aspect of the spermatic cord; the findings can be confirmed by genital imaging, and scrotal and transrectal ultrasound (TRUS). Unless associated with functional factors for infertility, patients with vasal aplasia will have normal testicular size and normal follicle-stimulating hormone (FSH), luteinizing hormone (LH), and testosterone levels.
Clinical Presentations and Diagnostic Imaging of VACTERL Association
Published in Fetal and Pediatric Pathology, 2023
Gabriele Tonni, Çağla Koçak, Gianpaolo Grisolia, Giuseppe Rizzo, Edward Araujo Júnior, Heron Werner, Rodrigo Ruano, Waldo Sepulveda, Maria Paola Bonasoni, Mario Lituania
In VACTERL, anatomical anomalies must occur between the 23rd and 56th day post-conception, as this embryological window is critical for the development of the vertebrae (23-32 days), heart, tracheoesophageal structures, forearm bones (29-41 days), and anorectal region (45-56 days). Malsegmentation of the vertebrae is the result of timing abnormalities in the segmentation clock. Anorectal and tracheoesophageal defects are due to disturbed mesodermal proliferation and migration, epithelial-mesenchymal interactions, and apoptosis. Radial aplasia can be caused by impairment in laying down, condensing, or chondrifying the angle of the radius. Renal and urinary tract anomalies may be the result of failed growth of the ureteric bud, metanephric mesenchyme, and mesonephros/mesonephric duct. Cardiac defects involve altered cardiac septal development, as atrioventricular septal defects and tetralogy of Fallot defects are common [13].
What are the key considerations for deciding on the use of CAR T-cell therapy for patients with follicular lymphoma?
Published in Expert Review of Anticancer Therapy, 2022
Beyond the improvement in efficacy compared to standard of care therapies, CAR T-cell therapy also offers the convenience of a ‘one and done’ mode of treatment delivery as opposed to either multiple cycles for 6 months or longer (ie cytotoxic chemotherapy), or therapies taken daily until progression of disease (ie PI3 kinase inhibitors and other small molecule inhibitors). These therapies may affect quality of life and activities of daily living for duration of treatment. The first month of CAR T-cell therapy can be accompanied by hospital admissions for the management of cytokine release syndrome or neurologic toxicity. Many elderly patients who experience grade 3 or higher neurologic toxicity become physically deconditioned or impaired to the point that even after resolution of neurologic toxicity, post-discharge rehabilitation at a skilled nursing facility may be required. Patients are unable to drive for 8 weeks after infusion of CAR T-cell therapy, and many patients therefore need significant social support during that time. Patients who lack social support or who are fiercely independent may opt for alternative therapies, reserving CAR T-cell therapy for future lines. Additionally, long-term toxicities include B-cell aplasia with accompanying hypogammaglobulinemia and infection risks.
Axicabtagene ciloleucel for the treatment of relapsed or refractory follicular lymphoma
Published in Expert Review of Anticancer Therapy, 2022
Axicabtagene ciloleucel was developed based on work conducted by James Kochenderfer and Steven A. Rosenberg at the Surgery Branch of the National Cancer Institute. In fact, the first patient successfully treated with CAR T-cell therapy using an axicabtagene ciloleucel construct had FL [52]. Published as a brief report in 2010, Kochenderfer et al. describe a male patient, initially diagnosed with grade 1, stage IVB FL in 2002 who was heavily pre-treated with PACE (prednisone, doxorubicin, cyclophosphamide, and etoposide), an idiotype vaccine, ipilimumab, and EPOCH-R (etoposide, prednisone, vincristine, cyclophosphamide, doxorubicin, and rituximab) prior to CAR T-cell treatment. After lymphocyte-depleting therapy, the patient received two consecutive days of CAR T-cell infusion with 1 × 108 cells on the first day and 3 × 108 cells on the following day. After the second infusion, he received 720,000 IU/kg of interleukin-2 every 8 hours for 8 doses. The patient achieved an impressive partial remission that lasted 32 weeks prior to progression. Treatment was accompanied by prolonged B cell aplasia, with evidence of early recovery at 36 weeks. The anti-CD19 CAR transgene was detected up to 27 weeks after CAR T-cell infusion.