Liječenje multiplog mijeloma kimeričnim T limfocitima

master's thesis

• Title (english): Treatment of multiple myeloma with chimeric T lymphocytes
• Author: Doris Čeović
• Mentor: Josip Batinić (mentor)
• Committee member: Nadira Duraković (predsjednik povjerenstva)
• Committee member: Marija Bakula (član povjerenstva)
• Committee member: Josip Batinić (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Internal Medicine); Zagreb
• Defense date and country: 2024-09-11, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract

Multipli mijelom (MM) je maligna bolest hematopoetskog sustava obilježena nekontroliranom klonalnom proliferacijom zloćudnih plazma stanica unutar koštane srži, prisutnošću monoklonalnog proteina u serumu i/ili urinu i pojavom sekundarnih oštećenja organa kao što su osteolitičke lezije, bubrežna insuficijencija, anemija i hiperkalcemija. To je druga najčešća hematološka neoplazma u visoko prihodnim zemljama. Čini 1-1,8% svih karcinoma s procijenjenom incidencijom u Europi 4,5-6,0/100000 godišnje. Unatoč napretku u terapiji, uključujući uporabu inhibitora proteasoma, imunomodulacijskih lijekova i monoklonskih protutijela, većina pacijenata uglavnom doživi recidiv i podlegne bolesti. Terapija kimeričnim T limfocitima pojavila se kao obećavajuće liječenje za relapsni i/ili refraktorni multipli mijelom. Ovaj inovativni pristup uključuje genetsku modifikaciju pacijentovih T-stanica kako bi se na njima izrazio CAR koji cilja specifične antigene na stanicama multiplog mijeloma, što dovodi do njihove destrukcije. Dva CAR-T lijeka, idekaptagen vikleucel i ciltakaptagen autoleucel, pokazala su izvanrednu učinkovitost u kliničkim ispitivanjima, postigavši duboke i trajne remisije kod teško pretretiranih pacijenata, te su odobrena od strane FDA i EMA.

Abstract (english)

Multiple myeloma (MM) is a malignant disease of the hematopoietic system characterized by uncontrolled clonal proliferation of malignant plasma cells within the bone marrow, the presence of a monoclonal protein in the serum/urine, and the occurrence of secondary end-organ damage such as osteolytic lesions, renal insufficiency, anemia, and hypercalcemia. It is the second most common hematological malignancy in high-income countries. It accounts for 1-1.8% of all cancers, with an estimated incidence in Europe of 4.5-6.0 per 100,000 annually. Despite advancements in therapy, including using proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies most patients eventually experience relapse and succumb to the disease. Chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a promising treatment for relapsed/refractory multiple myeloma. This innovative approach involves genetically modifying a patient’s T cells to express a CAR that targets specific antigens on myeloma cells, leading to their destruction. Two CAR-T products, idecabtagene vicleucel and ciltacabtagene autoleucel, have shown remarkable efficacy in clinical trials, achieving deep and durable remissions in heavily pretreated patients, and have been approved by the FDA and EMA.

• Keywords: idekaptagen vikleucel; imunoterapija; adoptivna; multipli mijelom
• Keywords (english): idecabtagene vicleucel; immunotherapy; adoptive; multiple myeloma
• Language: croatian
• URN:NBN: urn:nbn:hr:105:470958
• Study programme: Title: Medicine; Study programme type: university; Study level: integrated undergraduate and graduate; Academic / professional title: doktor/doktorica medicine (doktor/doktorica medicine)
• Type of resource: Text
• File origin: Born digital
• Access conditions: Open access
• Created on: 2024-09-08 16:39:48