• Title/Summary/Keyword: Hematologic Malignancies

Search Result 70, Processing Time 0.038 seconds

Unleashing the Therapeutic Potential of CAR-T Cell Therapy Using Gene-Editing Technologies

  • Jung, In-Young;Lee, Jungmin
    • Molecules and Cells
    • /
    • v.41 no.8
    • /
    • pp.717-723
    • /
    • 2018
  • Chimeric antigen receptor (CAR) T-cell therapy, an emerging immunotherapy, has demonstrated promising clinical results in hematological malignancies including B-cell malignancies. However, accessibility to this transformative medicine is highly limited due to the complex process of manufacturing, limited options for target antigens, and insufficient anti-tumor responses against solid tumors. Advances in gene-editing technologies, such as the development of Zinc Finger Nucleases (ZFNs), Transcription Activator-Like Effector Nucleases (TALENs), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9), have provided novel engineering strategies to address these limitations. Development of next-generation CAR-T cells using gene-editing technologies would enhance the therapeutic potential of CAR-T cell treatment for both hematologic and solid tumors. Here we summarize the unmet medical needs of current CAR-T cell therapies and gene-editing strategies to resolve these challenges as well as safety concerns of gene-edited CAR-T therapies.

Effects of Oral Care Protocol on Oral Mucositis in Hematologic Malignancy Patients Receiving Chemotherapy (구강관리 프로토콜 적용이 항암화학요법을 받는 혈액암 환자의 구내염에 미치는 영향)

  • Park, Soo Jin;Cha, Gyeong Suk;Kim, Hyeung Sun;Park, Eun Young
    • Journal of Korean Clinical Nursing Research
    • /
    • v.22 no.1
    • /
    • pp.1-9
    • /
    • 2016
  • Purpose: This study was done to identify effects of oral care protocol on oral mucositis and oral care performance in hematologic malignancy patients receiving chemotherapy. Methods: The design of this study was a nonequivalent control group pretest-posttest design. Both groups were patients diagnosed with hematologic malignancies who were receiving chemotherapy-each group had 20 patients. In the experimental group, patients were given intensive education on oral care based on the oral care protocol, whereas in the controlled group, each patient was given an educational brochure. Before chemotherapy, and 3 days, 7 days, and 14 days after chemotherapy, oral mucositis status of two groups were assessed using the guide to physical assessment of the oral cavity. Oral care performance was examined before chemotherapy and 14 days later. Results: The experimental group with the oral care protocol showed a significant difference (F=18.15, p<.001) in the oral mucositis status, and also in oral care performance (t=-10.33, p<.001). Conclusion: Findings indicate that the application of the oral care protocol is an effective tool for lowering the occurrence of oral mucositis and enhancing oral care performance in hematologic malignancy patients receiving chemotherapy.

Acute Surgical Abdomen in Childhood Malignancies (소아암환자에서 발생하는 급성 복증)

  • Park, Byung-Kwan;Moon, Suk-Bae;Jung, Sung-Eun;Jung, Kyu-Whan;Park, Kwi-Won
    • Advances in pediatric surgery
    • /
    • v.15 no.2
    • /
    • pp.103-112
    • /
    • 2009
  • Catheter related and perianal problems are common surgical complications encountered during the treatment of pediatric malignancies. However acute surgical abdominal emergencies are rare. The aim of this study is to review acute surgical abdominal complications that occur during the treatment of childhood malignancies. Out of a total of 1,222 patients who were newly diagnosed with malignant disease, between January 2003 and May 2008, there were 10 patients who required surgery because of acute abdominal emergencies. Their medical records were reviewed retrospectively. Hematologic malignancies were present in 7 patients (4 leukemia, 2 lymphoma, 1 Langerhans cell histiocytosis) and solid tumors in 3 patients (1 adrenocortical carcinoma, 1 desmoplastic small round cell tumor, 1 rhabdomyosarcoma). Seven patients had intestinal obstruction, two had gastrointestinal perforation and one, typhlitis. Intestinal obstructions were treated with resection of the involved segment with (N=2) or without (N=3) enterostomy. Two patients had enterostomy alone when resection could not be performed. Intestinal perforation was treated with primary repair. Typhlitis of the ascending colon was treated with ileostomy. Right hemicolectomy was necessary the next day because of the rapidly progressing sepsis. Three patients are now alive on chemotherapy and one patient was lost to followed-up. Among six patients who died, five died of their original disease progression and one of uncontrolled sepsis after intestinal perforation. Although rare, acute surgical abdominal complications can occur in childhood malignancies. Rapid and accurate diagnosis and appropriate operation are required for effective treatment of the complications.

  • PDF

Electrophysiological features and prognosis of peripheral neuropathy associated with IgM monoclonal gammopathy: a single-center analysis in South Korea

  • Sooyoung Kim;Bit Na Lee;Seung Woo Kim;Ha Young Shin
    • Annals of Clinical Neurophysiology
    • /
    • v.25 no.2
    • /
    • pp.84-92
    • /
    • 2023
  • Background: Clinical spectrum of immunoglobulin M (IgM) monoclonal gammopathy varies from IgM monoclonal gammopathy of unknown significance (IgM-MGUS) to hematological malignancies. We evaluated the clinical features, electrophysiological characteristics, and prognosis of patients with peripheral neuropathy associated with IgM monoclonal gammopathy (PN-IgM MG). Methods: We retrospectively evaluated 25 patients with PN-IgM MG. Peripheral neuropathy was classified as axonal, demyelinating, or undetermined, based on electrophysiological studies. We classified the enrolled patients into the IgM-MGUS and malignancy groups, and compared the clinical and electrophysiological features between the groups. Results: Fifteen patients had IgM-MGUS and 10 had hematologic malignancies (Waldenström's macroglobulinemia: two and B-cell non-Hodgkin's lymphoma: eight). In the electrophysiological evaluation, the nerve conduction study (NCS) criteria for demyelination were met in 86.7% of the IgM-MGUS group and 10.0% of the malignancy group. In particular, the distal latencies of the motor NCS in the IgM-MGUS group were significantly prolonged compared to those in the malignancy group (median, 9.1 ± 5.1 [IgM-MGUS], 4.2 ± 1.3 [malignancy], p = 0.003; ulnar, 5.4 ± 1.9 [IgM-MGUS], 2.9 ± 0.9 [malignancy], p = 0.001; fibular, 9.3 ± 5.1 [IgM-MGUS], 3.8 ± 0.3 [malignancy], p = 0.01; P-posterior tibial, 8.3 ± 5.4 [IgM-MGUS], 4.4 ± 1.0 [malignancy], p = 0.04). Overall treatment responses were significantly worse in the malignancy group than in the IgM-MGUS group (p = 0.004), and the modified Rankin Scale score at the last visit was higher in the malignancy group than in the IgM-MGUS group (2.0 ± 1.1 [IgM-MGUS], 4.2 ± 1.7 [malignancy], p = 0.001), although there was no significant difference at the initial assessment. Conclusions: The risk of hematological malignancy should be carefully assessed in patients with PN-IgM MG without electrophysiological demyelination features.

Comparing Perceptions, Determinants, and Needs of Patients, Family Members, Nurses, and Physicians When Making Life-Sustaining Treatment Decisions for Patients with Hematologic Malignancies

  • Kim, Semi;Ham, Eun Hye;Kim, Dong Yeon;Jang, Seung Nam;Kim, Min kyeong;Choi, Hyun Ah;Cho, Yun A;Lee, Seung A;Yun, Min Jeong
    • Journal of Hospice and Palliative Care
    • /
    • v.25 no.1
    • /
    • pp.12-24
    • /
    • 2022
  • Purpose: This descriptive study compared the perceptions, determinants, and needs of patients, family members, nurses, and physicians regarding life-sustaining treatment decisions for patients with hematologic malignancies in the hematology-oncology department of a tertiary hospital in Seoul, Korea. Methods: In total, 147 subjects were recruited, gave written consent, and provided data by completing a structured questionnaire. Data were analyzed using analysis of variance, the chi-square test, and the Fisher exact test. Results: Nurses (F=3.35) and physicians (F=3.57) showed significantly greater familiarity with the Act on Decisions on Life-Sustaining Treatment than patients (F=2.69) and family members (F=2.59); (F=19.58, P<0.001). Many respondents, including 19 (51.4%) family members, 16 (43.2%) physicians, and 11 (29.7%) nurses, agreed that the patient's opinion had the greatest effect when making life-sustaining treatment decisions. Twelve (33.3%) patients answered that mental, physical, and financial burdens were the most important factors in life-sustaining treatment decisions, and there was a significant difference among the four groups (P<0.001). Twenty-four patients (66.7%), 27 (73.0%) family members, and 21(56.8%) nurses answered that physicians were the most appropriate people to provide information regarding life-sustaining treatment decisions. Unexpectedly, 19 (51.4%) physicians answered that hospice nurse practitioners were the most appropriate people to talk to about life-sustaining treatment (P<0.001). Conclusion: It is of utmost importance that the patient and physician determine when life-sustaining treatment should be withdrawn, with the patient making the ultimate decision. Doctors and nurses have the responsibility to provide detailed information. The goal of end-of-life planning is to ensure patients' dignity and respect their values.

Leukoencephalopathy after CNS Prophylactic Therapy in Pediatric Hematologic Malignancy (소아 혈액종양 환자에서 중추신경계 예방적 치료 후 발생한 백질뇌병증)

  • Lee, Jun Hwa;Lee, Sun Min;Choi, Eun Jin;Lee, Kun Soo
    • Clinical and Experimental Pediatrics
    • /
    • v.46 no.6
    • /
    • pp.566-571
    • /
    • 2003
  • Purpose : Leukoencephalopathy(LE) is one of the most serious complications in children with hematologic malignancies during the course of treatment. Early recognition is important to reduce the impact and sequelae from LE. We therefore investigated the clinical features of LE following central nervous system(CNS) prophylaxis in children with hematologic malignancies and evaluated the significance of regular check-ups of brain MRI. Methods : We retrospectively reviewed children with hematologic malignancies who had CNS prophylaxis including intrathecal(IT) methotrexate(MTX) and/or cranial irradiation at the Department of Pediatrics, Kyungpook National University Hospital from Oct. 1995 to May 2002. Fifteen cases of acute leukemia and one case of lymphoma who experienced LE following CNS prophylaxis were included in the study. Clinical data were analyzed from the medical records and brain MRIs were reviewed by neuroradiologists. Results : The ages ranged from 1 to 13 years(median age=5.2 years), and the male to female ratio was 3 : 1. The time interval from the beginning of chemotherapy to the time of diagnosis of LE ranged from 2 to 17 months. They all had IT MTX two to 15 times and ten underwent cranial irradiation(1,800 rads). At the time of diagnosis, ten of them had neuropsychiatric symptoms including seizures, personality changes, headache, etc. After the change of treatment modality, four cases showed significant improvement on follow-up MRIs, six cases had no significant changes and two had worsening of LE. Four patients died of infection and bone marrow relapse. Conclusion : CNS prophylaxis with IT therapy and cranial irradiation may cause leukoencephalopathy during the course of treatment. As a result, regular brain MRI check-up is recommended for the early detection and reducing the incidence of LE, along with changes in the treatment modality.

A Novel Translocation Involving RUNX1 and HOXA Gene Clusters in a Case of Acute Myeloid Leukemia with t(7;21)(p15;q22)

  • Moon, Yeonsook;Horsman, Douglas E.;Humphries, R. Keith;Park, Gyeongsin
    • IMMUNE NETWORK
    • /
    • v.13 no.5
    • /
    • pp.222-226
    • /
    • 2013
  • Translocations involving chromosome 21q22 are frequently observed in hematologic malignancies including acute myeloid leukemia (AML), most of which have been known to be involved in malignant transformation through transcriptional dysregulation of Runt-related transcription factor 1 (RUNX1) target genes. Nineteen RUNX1 translocational partner genes, at least, have been identified, but not Homeobox A (HOXA) genes so far. We report a novel translocation of RUNX1 into the HOXA gene cluster in a 57-year-old female AML patient who had been diagnosed with myelofibrosis 39 months ahead. G-banding showed 46,XX,t(7;21)(p15;q22). The involvement of RUNX1 and HOXA genes was confirmed by fluorescence in situ hybridization.

A Case of Capecitabine-Induced Sarcoidosis

  • Kang, Shin-Myung;Baek, Ji-Yeon;HwangBo, Bin;Kim, Hyae-Young;Lee, Geon-Kook;Lee, Hee-Seok
    • Tuberculosis and Respiratory Diseases
    • /
    • v.72 no.3
    • /
    • pp.318-322
    • /
    • 2012
  • Sarcoidosis is an inflammatory disease involving multiple-organs with an unknown cause. The new onset of sarcoidosis associated with therapeutic agents has been observed in 3 clinical settings; tumor necrosis factor antagonists in autoimmune rheumatologic diseases, interferon alpha with or without ribavirin in patients with chronic hepatitis C or melanoma, and antineoplastic agent-associated sarcoidosis in patients with hematologic malignancies. Here, we report a female patient who developed sarcoidosis after capecitabine treatment as an adjuvant chemotherapy for sigmoid colon cancer. To our knowledge, this is the first report of a capecitabine-induced sarcoidosis.

Eosinophil disorders (호산구이상증)

  • Kim, Sun Young
    • Clinical and Experimental Pediatrics
    • /
    • v.52 no.6
    • /
    • pp.643-648
    • /
    • 2009
  • Blood eosinophilia can be classified as either familial or acquired. Familial eosinophilia is a rare autosomal dominant disorder characterized by a stable eosinophil count. Acquired eosinophilia is classified further into a primary or secondary phenomenon depending on whether eosinophils are considered integral to the underlying disease. Primary eosinophilia is considered clonal in the presence of either a cytogenetic abnormality or bone marrow histological evidence of classified hematologic malignancies. Causes of secondary eosinophilia include infections, allergic or immunologic disorders, and drugs. Idiopathic eosinophilia belongs to a category of primary eosinophilia, and this is a diagnosis of exclusion. Cases with eosinophilia that lack evidence of clonality may be diagnosed as idiopathic hypereosinophilic syndrome after all causes of reactive eosinophilia have been eliminated. Genetic mutations involving the platelet-derived growth receptor genes (PDGFRA and PDGFRB) have been pathogenetically linked to clonal eosinophilia, and their presence predicts the treatment response to imatinib. In this review, I will present a clinical summary of both familial and acquired eosinophilia with emphasis on recent developments in molecular pathogenesis and treatment.