Učinkovitost poslijetransplantacijskog ciklofosfamida u prevenciji bolesti darivatelja protiv primatelja nakon alogenične transplantacije krvotvornih matičnih stanica od nesrodnog nepodudarnog darivatelja

master's thesis

• Title (english): Efficacy of post-transplant cyclophosphamide in the prevention of graft-versus-host disease following allogeneic hematopoietic stem cell transplant from an unrelated mismatched donor
• Author: Lea Jukić
• Mentor: Nadira Duraković (mentor)
• Committee member: Josip Batinić (predsjednik povjerenstva)
• Committee member: Martina Lovrić Benčić (član povjerenstva)
• Granter: University of Zagreb; School of Medicine; (Department of Internal Medicine); Zagreb
• Defense date and country: 2024-07-12, Croatia
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Clinical Medical Sciences; Internal Medicine

Abstract

Transplantacija hematopoetskih matičnih stanica potencijalno je kurativna metoda liječenja različitih hematoloških i nehematoloških bolesti. Za život opasna bolest darivatelja protiv primatelja jedna je od glavnih komplikacija transplantacije hematopoetskih matičnih stanica. Poslijetransplantacijski ciklofosfamid (PTCy) uspješno se koristi kao profilaksa bolesti darivatelja protiv primatelja (GVHD) nakon haploidentične transplantacije hematopoetskih matičnih stanica, a istraživanja pokazuju kako se njegovo profilaktično djelovanje očituje i u alogeničnoj transplantaciji, premošćujući HLA-barijeru. U svrhu ispitivanja učinkovitosti poslijetransplantacijskog ciklofosfamida nakon alogenične transplantacije od nesrodnog nepodudarnog darivatelja provedena je retrospektivna studija primatelja alogenične transplantacije KBC-a Zagreb između siječnja 2020. te lipnja 2023. Izdvojen je 161 pacijent, od kojih je je 120 primilo transplantaciju od nesrodnog podudarnog darivatelja, a 41 od nesrodnog nepodudarnog darivatelja. 21 pacijent primio je transplantaciju od nesrodnog nepodudarnog darivatelja te PTCy. Uspoređeni su parametri GVHD-a između nepodudarne skupine koja je primila PTCy te nepodudarne (nePTCy 9/10) i podudarne (nePTCy 10/10) skupine koje nisu primile PTCy. Sveukupni udio slučajeva akutnog GVHD-a (aGVHD-a) bio je najniži u skupini pacijenata koji su primili PTCy (n=4, 19%), u odnosu na nepodudarnu (n=8, 40%) i podudarnu nePTCy skupinu (nePTCy 10/10, n=41, 34,5%). Sveukupni udio slučajeva kroničnog GVHD-a (cGVHD-a) bio je najniži u podudarnoj nePTCy skupini (n=21, 17,65%), zatim u PTCy skupini (n=4, 19%) te u nepodudarnoj nePTCy skupini (n=4, 20%). Ovim istraživanjem dobiveni su preliminarni podaci koji upućuju na potencijalno zaštitno djelovanje PTCy u profilaksi GVHD-a, međutim ne postiže se statistička značajnost (p>0.05) te je potrebno daljnje istraživanje s više ispitanika te podrobnija statistička analiza kako bi se potvrdili ovi nalazi.

Abstract (english)

Hematopoietic stem cell transplantation is a potentially curative treatment method for various hematologic and non-hematologic diseases. The life-threatening graft-versus-host disease is one of the main complications of hematopoietic stem cell transplantation. Post-transplantation cyclophosphamide (PTCy) is successfully used as graft-versus-host disease (GVHD) prophylaxis after haploidentical stem cell transplantation, but research shows its effect in allogeneic stem cell transplantation, crossing the HLA-barrier. To examine the efficacy of post-transplantation cyclophosphamide after allogeneic stem cell transplantation from a mismatched unrelated donor, a retrospective study was conducted on allogeneic stem cell transplantation recipients between January 2020 and June 2023 at the University Hospital Center Zagreb. 161 patients were identified, from which 120 received transplantation from a matched unrelated donor, and 41 from a mismatched unrelated donor. 21 patients received an allogeneic stem cell transplantation from a mismatched unrelated donor and also received PTCy. GVHD parameters were compared between the mismatched group that received PTCy and the mismatched (nonPTCy 9/10) and matched (nonPTCy 10/10) groups that have not received PTCy. The overall proportion of acute GVHD cases was the lowest in the group that received PTCy (n=4, 19%), in comparison to the mismatched (n=8, 40%) and matched nonPTCy groups (n=41, 34,5%). The overall proportion of chronic GVHD cases was the lowest in the matched nonPTCy group (n=21, 17,65%), followed by the PTCy group (n=4, 19%) and the mismatched nonPTCy group (n=4, 20%). This study yielded preliminary findings suggesting a potentially protective effect of PTCy as GVHD prophylaxis, but without reaching statistical significance (p>0.05). Further research with more subjects and more thorough statistical analysis is needed to confirm these findings.

• Keywords: ciklofosfamid; GVHD; transplantacija hematopoetskih matičnih stanica; nepodudarni donori
• Keywords (english): Cyclophosphamide; Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Unrelated Donors
• Language: croatian
• URN:NBN: urn:nbn:hr:105:438730
• 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: 2024-07-02 19:54:36