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  1. Article: Influence of Acute and Chronic Graft-Versus-Host Disease on Persistence of Antibodies against Measles, Mumps, Rubella and Varicella in the First Year after Autologous or Allogeneic Hematopoietic Stem Cell Transplantation.

    Harrison, Nicole / Burgmann, Heinz / Rabitsch, Werner / Honsig, Claudia / Robak, Oliver

    Vaccines

    2023  Volume 11, Issue 3

    Abstract: Patients after hematopoietic stem cell transplantation (HSCT) are vulnerable to infections due to severe immunosuppression. Live-attenuated vaccines are contraindicated for two years after HSCT. The aim of this study was to assess the persistence of ... ...

    Abstract Patients after hematopoietic stem cell transplantation (HSCT) are vulnerable to infections due to severe immunosuppression. Live-attenuated vaccines are contraindicated for two years after HSCT. The aim of this study was to assess the persistence of antibodies against measles, mumps, rubella and varicella in the first year after HSCT. Forty patients undergoing autologous (
    Language English
    Publishing date 2023-03-14
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2703319-3
    ISSN 2076-393X
    ISSN 2076-393X
    DOI 10.3390/vaccines11030656
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Cytokine Release Syndrome during Antithymocyte Globulin/Anti-T Lymphocyte Globulin Serotherapy for Graft-versus-Host Disease Prophylaxis before Allogeneic Hematopoietic Stem Cell Transplantation: Incidence and Early Clinical Impact According to American Society of Transplantation and Cellular Therapy Grading Criteria.

    Knaus, Hanna A / Rottner, Theresa / Baumann, Clara K / Cserna, Julia / Mitterbauer, Margit / Schulenburg, Axel / Rabitsch, Werner / Wohlfarth, Philipp

    Transplantation and cellular therapy

    2022  Volume 28, Issue 5, Page(s) 260.e1–260.e9

    Abstract: Antithymocyte globulin (ATG)/anti-T lymphocyte globulin (ATLG) aids graft-versus-host disease (GVHD) prophylaxis in HLA-matched related and unrelated donor hematopoietic stem cell transplantation (HSCT). Its use is frequently accompanied by systemic ... ...

    Abstract Antithymocyte globulin (ATG)/anti-T lymphocyte globulin (ATLG) aids graft-versus-host disease (GVHD) prophylaxis in HLA-matched related and unrelated donor hematopoietic stem cell transplantation (HSCT). Its use is frequently accompanied by systemic infusion reactions attributable to cytokine release syndrome (CRS). However, detailed data on ATG/ATLG-induced CRS and its correlation with clinical outcome parameters are lacking. This study aimed to analyze the incidence, characteristics, risk factors, and early clinical impact of CRS during ATG/ATLG administration before allogeneic HSCT according to the American Society of Transplantation and Cellular Therapy (ASTCT) CRS grading criteria. This retrospective single-center analysis included consecutive recipients of allogeneic HSCT treated with ATG/ATLG as GVHD prophylaxis at the Medical University of Vienna between January 1, 2014, and August 15, 2021. Multivariate regression models were used to explore risk factors for CRS and its association with clinical outcomes (acute GVHD grade II-IV, clinically significant cytomegalovirus infection, nonrelapse mortality, and overall survival) at 6 months after HSCT. A total of 284 patients (median age, 54 years; interquartile range [IQR], 45 to 61 years; 120 females, 164 males) were included in the study. ATLG was used in 222 patients (78%); ATG, in 62 (22%). One hundred sixty-six patients (58%) developed CRS grade ≥1 during ATG/ATLG administration. CRS was mostly mild, with 92% of the cases CRS grade 1-2. Thirteen patients (5%) developed CRS grade 3, and 1 patient had CRS grade 4. No CRS-related death (grade 5) occurred. Patients with CRS showed a pronounced systemic inflammatory response as measured by inflammatory markers C-reactive protein, IL-6, and procalcitonin. In multivariate analysis, lymphoma as the underlying disease, high ATLG dose of 60 mg/kg, and body weight were significantly associated with CRS. Patients with CRS grade ≥1 had a higher 6-month incidence of acute GVHD II-IV compared with patients without CRS (24% versus 14%; P = .04). This effect remained statistically significant only for CRS grade 3-4 (subdistribution hazard ratio, 3.70; 95% confidence interval, 1.58 to 8.68; P < .01) after adjusting for relevant confounders. Other clinical outcome parameters were not affected by the occurrence of CRS. In our cohort, CRS defined by ASTCT grading was a frequent but mostly mild complication following ATG/ATLG administration for GVHD prophylaxis. Our data suggest a possible interaction of (higher-grade) CRS with an increased risk for developing acute GVHD. Further studies to corroborate this finding are warranted, as it could inform the investigation of additional prophylactic interventions, such as IL-6 blockade, in this setting.
    MeSH term(s) Antibodies ; Antilymphocyte Serum/therapeutic use ; Cytokine Release Syndrome ; Female ; Graft vs Host Disease/epidemiology ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Immunization, Passive/adverse effects ; Incidence ; Interleukin-6 ; Male ; Middle Aged ; Retrospective Studies
    Chemical Substances Antibodies ; Antilymphocyte Serum ; Interleukin-6
    Language English
    Publishing date 2022-02-22
    Publishing country United States
    Document type Journal Article
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2022.02.016
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  3. Article ; Online: Antibody-Based and Cell Therapies for Advanced Mastocytosis: Established and Novel Concepts.

    Valent, Peter / Akin, Cem / Arock, Michel / Gleixner, Karoline V / Greinix, Hildegard / Hermine, Olivier / Horny, Hans-Peter / Ivanov, Daniel / Orfao, Alberto / Rabitsch, Werner / Reiter, Andreas / Schulenburg, Axel / Sotlar, Karl / Sperr, Wolfgang R / Ustun, Celalettin

    International journal of molecular sciences

    2023  Volume 24, Issue 20

    Abstract: Advanced systemic mastocytosis (SM) is a heterogeneous group of myeloid neoplasms characterized by an uncontrolled expansion of mast cells (MC) in one or more internal organs, SM-induced tissue damage, and poor prognosis. Advanced SM can be categorized ... ...

    Abstract Advanced systemic mastocytosis (SM) is a heterogeneous group of myeloid neoplasms characterized by an uncontrolled expansion of mast cells (MC) in one or more internal organs, SM-induced tissue damage, and poor prognosis. Advanced SM can be categorized into aggressive SM (ASM), MC leukemia (MCL), and SM with an associated hematologic neoplasm (SM-AHN). In a vast majority of all patients, neoplastic cells display a
    MeSH term(s) Humans ; Quality of Life ; Mastocytosis/genetics ; Mastocytosis/therapy ; Mastocytosis, Systemic/therapy ; Mastocytosis, Systemic/drug therapy ; Mast Cells ; Mutation ; Proto-Oncogene Proteins c-kit/genetics
    Chemical Substances Proto-Oncogene Proteins c-kit (EC 2.7.10.1)
    Language English
    Publishing date 2023-10-12
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2019364-6
    ISSN 1422-0067 ; 1422-0067 ; 1661-6596
    ISSN (online) 1422-0067
    ISSN 1422-0067 ; 1661-6596
    DOI 10.3390/ijms242015125
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  4. Article ; Online: Diverse macrophage populations contribute to distinct manifestations of human cutaneous graft-versus-host disease.

    Strobl, Johanna / Gail, Laura M / Krecu, Laura / Madad, Shaista / Kleissl, Lisa / Unterluggauer, Luisa / Redl, Anna / Brazdilova, Kveta / Saluzzo, Simona / Wohlfarth, Philipp / Knaus, Hanna A / Mitterbauer, Margit / Rabitsch, Werner / Haniffa, Muzlifah / Stary, Georg

    The British journal of dermatology

    2023  Volume 190, Issue 3, Page(s) 402–414

    Abstract: Background: Graft-versus-host disease (GvHD) is a major life-threatening complication of allogeneic haematopoietic stem cell transplantation (HSCT), limiting the broad application of HSCT for haematological malignancies. Cutaneous GvHD is described as a ...

    Abstract Background: Graft-versus-host disease (GvHD) is a major life-threatening complication of allogeneic haematopoietic stem cell transplantation (HSCT), limiting the broad application of HSCT for haematological malignancies. Cutaneous GvHD is described as a post-transplant inflammatory reaction by skin-infiltrating donor T cells and remaining recipient tissue-resident memory T cells. Despite the major influence of lymphocytes on GvHD pathogenesis, the complex role of mononuclear phagocytes (MNPs) in tissues affected by GvHD is increasingly appreciated.
    Objectives: To characterize the identity, origin and functions of MNPs in patients with acute cutaneous GvHD.
    Methods: Using single-cell RNA sequencing and multiplex tissue immunofluorescence, we identified an increased abundance of MNPs in skin and blood from 36 patients with acute cutaneous GvHD. In cases of sex-mismatched transplantation, we used expression of X-linked genes to detect rapid tissue adaptation of newly recruited donor MNPs resulting in similar transcriptional states of host- and donor-derived macrophages within GvHD skin lesions.
    Results: We showed that cutaneous GvHD lesions harbour expanded CD163+ tissue-resident macrophage populations with anti-inflammatory and tissue-remodelling properties including interleukin-10 cytokine production. Cell-cell interaction analyses revealed putative signalling to strengthen regulatory T-cell responses. Notably, macrophage polarization in chronic cutaneous GvHD types was proinflammatory and drastically differed from acute GvHD, supporting the notion of distinct cellular players in different clinical GvHD subtypes.
    Conclusions: Overall, our data reveal a surprisingly dynamic role of MNPs after HSCT. Specific and time-resolved targeting to repolarize this cell subset may present a promising therapeutic strategy in combatting GvHD skin inflammation.
    MeSH term(s) Humans ; Graft vs Host Disease/pathology ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Macrophages/metabolism ; Skin Diseases/pathology ; Cytokines
    Chemical Substances Cytokines
    Language English
    Publishing date 2023-11-17
    Publishing country England
    Document type Journal Article
    ZDB-ID 80076-4
    ISSN 1365-2133 ; 0007-0963
    ISSN (online) 1365-2133
    ISSN 0007-0963
    DOI 10.1093/bjd/ljad402
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  5. Article ; Online: The assessment of platelet function by thromboelastometry as a point-of-care test to guide Intercept-treated platelet support in hemato-oncological patients and hematopoietic stem cell transplantation recipients.

    Leitner, Gerda C / Ho, Markus / Tolios, Alexander / Hopfinger, Georg / Rabitsch, Werner / Wohlfarth, Philipp

    Transfusion

    2020  Volume 60, Issue 7, Page(s) 1391–1399

    Abstract: Background: Pathogen inactivation (PI) techniques for platelet concentrates (PCs) are one of the latest innovations to improve blood safety and reduce the risk of transfusion-transmitted infections (TTIs). An impaired function and in vivo recovery of ... ...

    Abstract Background: Pathogen inactivation (PI) techniques for platelet concentrates (PCs) are one of the latest innovations to improve blood safety and reduce the risk of transfusion-transmitted infections (TTIs). An impaired function and in vivo recovery of platelets as well as an increased PC demand are concerns regarding these techniques. The intent of this study was to evaluate the hemostatic effect of PCs treated with the Intercept™ System by thromboelastometry (TEM) and to assess the clinical validity of its results in comparison to post-transfusion increase (PTI) and corrected count increment (CCI).
    Study-design and methods: This prospective-observational study included 47 patients (m:f = 25:22; median age: 54 years [21-70]) of our Bone Marrow Transplantation unit with hemato-oncological malignancies transfused with Intercept™-treated PCs. Serial TEM measurements were performed just before and 1 hour after PC transfusion and were analyzed for their correlation with PTI and CCI as well as for clinical variables.
    Results: The majority of our patients had received a hematopoietic stem cell transplantation (HSCT) (n = 41; 87%). In median 9 (1-50) PCs were transfused. Serial TEM, PTI, and CCI measurements were available for 150 transfusion episodes. The median platelet dose transfused was 2.65 × 10
    Conclusion: Serial TEM measurements indicate the hemostatic effect of Intercept™-treated PCs. The 1-hour PTI and CCI may not appropriately reflect the in vivo function of platelets after PI PC transfusion.
    MeSH term(s) Adult ; Aged ; Allografts ; Blood Platelets/metabolism ; Female ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Middle Aged ; Neoplasms/blood ; Neoplasms/therapy ; Platelet Transfusion ; Point-of-Care Testing ; Prospective Studies ; Thrombelastography
    Language English
    Publishing date 2020-04-22
    Publishing country United States
    Document type Clinical Trial ; Journal Article ; Observational Study
    ZDB-ID 208417-x
    ISSN 1537-2995 ; 0041-1132
    ISSN (online) 1537-2995
    ISSN 0041-1132
    DOI 10.1111/trf.15783
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  6. Article ; Online: Emergency Department Utilization Following Allogeneic Hematopoietic Stem Cell Transplantation: A Single-Center Retrospective Longitudinal Analysis of 557 Patients.

    Cserna, Julia / Baumann, Clara K / Lobmeyr, Elisabeth / Grafeneder, Juergen / Ettl, Florian / Eibensteiner, Felix / Rabitsch, Werner / Mitterbauer, Margit / Knaus, Hanna A / Wohlfarth, Philipp

    Transplantation and cellular therapy

    2023  Volume 29, Issue 5, Page(s) 321.e1–321.e9

    Abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) recipients are at risk of various complications during post-transplantation follow-up. Some patients may refer to an emergency department (ED) for medical attention, but data on ED visits by HSCT ... ...

    Abstract Allogeneic hematopoietic stem cell transplantation (HSCT) recipients are at risk of various complications during post-transplantation follow-up. Some patients may refer to an emergency department (ED) for medical attention, but data on ED visits by HSCT recipients are lacking. In the present study, we aimed to assess ED utilization in HSCT recipients and associated risk factors during post-transplantation follow-up, identify subgroups of HSCT recipients presenting to the ED, analyze outcomes and prognostic factors for hospitalization and 30-day mortality after ED visits, and assess mortality hazard following an ED presentation. The study involved a retrospective single-center longitudinal analysis including 557 consecutive recipients of allogeneic HSCT at the Medical University of Vienna, Austria, between January 2010 and January 2020. Descriptive statistics, event estimates accounting for censored data with competing risks, latent class analysis, and multivariate regression models were used for data analysis. Out of 557 patients (median age at HSCT, 49 years [interquartile range (IQR), 39 to 58 years]; 233 females and 324 males), 137 (25%) presented to our center's ED at least once during post-HSCT follow-up (median individual follow-up, 2.66 years; IQR, .72 to 5.59 years). Cumulative incidence estimates of a first ED visit in the overall cohort were 19% at 2 years post-HSCT, 25% at 5 years post-HSCT, and 28% at 10 years post-HSCT. These estimates were increased to 34%, 41%, and 43%, respectively, in patients residing in Vienna. Chronic graft-versus-host disease (GVHD) was the sole risk factor showing a statistically significant association with ED presentation in multivariate analysis (hazard ratio [HR], 2.34; 95% confidence interval [CI], 1.63 to 3.35). Patients presented to the ED with various and often multiple symptoms. We identified 3 latent patient groups in the ED, characterized mainly by the time from HSCT, chronic GVHD, and documented pulmonary infection. Hospitalization was required in 132 of all 216 analyzed ED visits (61%); in-hospital mortality and 30-day mortality rates were 13% and 7%, respectively. Active acute GVHD, systemic steroids, documented infection, pulmonary infiltrates, and oxygen supplementation were statistically significant predictors of hospitalization; shorter time from HSCT, pulmonary infiltrates, and hemodynamic instability were independent risk factors for 30-day mortality. ED presentation during the last 30 days increased the mortality hazard in the overall cohort (HR, 4.56; 95% CI, 2.68 to 7.76) after adjustment for relevant confounders. One-quarter of the patients visited the ED for medical attention at least once during post-HSCT follow-up. Depending on the presence of identified risk factors, a significant proportion of patients may require hospitalization and be at risk for adverse outcomes. Screening for these risk factors and specialist consultation should be part of managing most HSCT recipients presenting to the ED.
    MeSH term(s) Male ; Female ; Humans ; Middle Aged ; Retrospective Studies ; Transplantation, Homologous/adverse effects ; Graft vs Host Disease/epidemiology ; Graft vs Host Disease/etiology ; Risk Factors ; Hematopoietic Stem Cell Transplantation/adverse effects
    Language English
    Publishing date 2023-02-24
    Publishing country United States
    Document type Journal Article
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2023.02.018
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  7. Article ; Online: A bi-centric experience of extracorporeal carbon dioxide removal (ECCO

    Wohlfarth, Philipp / Schellongowski, Peter / Staudinger, Thomas / Rabitsch, Werner / Hermann, Alexander / Buchtele, Nina / Turki, Amin T / Tzalavras, Asterios / Liebregts, Tobias

    Artificial organs

    2021  Volume 45, Issue 8, Page(s) 903–910

    Abstract: Acute respiratory failure (ARF) is the main reason for ICU admission following allogeneic hematopoietic stem cell transplantation (HSCT). Extracorporeal ... ...

    Abstract Acute respiratory failure (ARF) is the main reason for ICU admission following allogeneic hematopoietic stem cell transplantation (HSCT). Extracorporeal CO
    MeSH term(s) Adult ; Blood Gas Analysis ; Extracorporeal Membrane Oxygenation/methods ; Female ; Graft vs Host Disease ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Middle Aged ; Prognosis ; Respiratory Insufficiency/etiology ; Respiratory Insufficiency/therapy ; Retrospective Studies
    Language English
    Publishing date 2021-05-04
    Publishing country United States
    Document type Journal Article ; Multicenter Study
    ZDB-ID 441812-8
    ISSN 1525-1594 ; 0160-564X
    ISSN (online) 1525-1594
    ISSN 0160-564X
    DOI 10.1111/aor.13931
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  8. Article ; Online: Vienna Cancer Stem Cell Club (VCSCC): 20 year jubilee and future perspectives.

    Valent, Peter / Sadovnik, Irina / Peter, Barbara / Ivanov, Daniel / Schulenburg, Axel / Hadzijusufovic, Emir / Willmann, Michael / Rülicke, Thomas / Herrmann, Harald / Rabitsch, Werner / Karlic, Heidrun / Gleixner, Karoline V / Sperr, Wolfgang R / Hoermann, Gregor / Dahlhoff, Maik / Pfeilstöcker, Michael / Keil, Felix / Lion, Thomas / Grunt, Thomas W

    Expert review of hematology

    2023  Volume 16, Issue 9, Page(s) 659–670

    Abstract: Introduction: The Vienna Cancer Stem Cell Club (VCSCC) was launched by a group of scientists in Vienna in 2002.: Areas covered: Major aims of the VCSCC are to support research on cancer stem cells (CSC) in hematopoietic malignancies and to translate ... ...

    Abstract Introduction: The Vienna Cancer Stem Cell Club (VCSCC) was launched by a group of scientists in Vienna in 2002.
    Areas covered: Major aims of the VCSCC are to support research on cancer stem cells (CSC) in hematopoietic malignancies and to translate CSC-related markers and targets into clinical application. A primary focus of research in the VCSCC is the leukemic stem cell (LSC). Between 2013 and 2021, members of the VCSCC established a special research program on myeloproliferative neoplasms and since 2008, members of the VCSCC run the Ludwig Boltzmann Institute for Hematology and Oncology. In all these years, the VCSCC provided a robust intellectual platform for translational hematology and LSC research in Vienna. Furthermore, the VCSCC interacts with several national and international study groups and societies in the field. Representatives of the VCSCC also organized a number of international meetings and conferences on neoplastic stem cells, including LSC, in the past 15 years, and contributed to the definition and classification of CSC/LSC and related pre-malignant and malignant conditions.
    Expert opinion: The VCSCC will continue to advance the field and to develop LSC-detecting and LSC-eradicating concepts through which diagnosis, prognostication, and therapy of blood cancer patients should improve.
    MeSH term(s) Humans ; Leukemia, Myelogenous, Chronic, BCR-ABL Positive/drug therapy ; Neoplastic Stem Cells/pathology ; Hematologic Neoplasms/pathology ; Forecasting
    Language English
    Publishing date 2023-07-27
    Publishing country England
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 2516804-6
    ISSN 1747-4094 ; 1747-4086
    ISSN (online) 1747-4094
    ISSN 1747-4086
    DOI 10.1080/17474086.2023.2232545
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  9. Article ; Online: Epigenetic regulation of T cell lineages in skin and blood following hematopoietic stem cell transplantation.

    Pandey, Ram Vinay / Strobl, Johanna / Redl, Anna / Unterluggauer, Luisa / Gail, Laura / Kleissl, Lisa / Müller, Sophie / Atzmüller, Denise / Fife-Gernedl, Victoria / Krausgruber, Thomas / Knaus, Hanna / Mitterbauer, Margit / Wohlfarth, Philipp / Rabitsch, Werner / Bock, Christoph / Stary, Georg

    Clinical immunology (Orlando, Fla.)

    2023  Volume 248, Page(s) 109245

    Abstract: Allogeneic hematopoietic stem-cell transplantation (HSCT) seeks to reconstitute the host's immune system from donor stem cells. The success of HSCT is threatened by complications including leukemia relapse or graft-versus-host-disease (GvHD). To ... ...

    Abstract Allogeneic hematopoietic stem-cell transplantation (HSCT) seeks to reconstitute the host's immune system from donor stem cells. The success of HSCT is threatened by complications including leukemia relapse or graft-versus-host-disease (GvHD). To investigate the underlying regulatory processes in central and peripheral T cell recovery, we performed sequential multi-omics analysis of T cells of the skin and blood during HSCT. We detected rapid effector T cell reconstitution, while emergence of regulatory T cells was delayed. Epigenetic and gene-regulatory programs were associated with recovering T cells and diverged greatly between skin and blood T cells. The BRG1/BRM-associated factor chromatin remodeling complex and histone deacetylases (HDACs) were epigenetic regulators involved in restoration of T cell homeostasis after transplantation. In isolated T cells of patients after HSCT, we observed class I HDAC-inhibitors to modulate their dysbalance. The present study highlights the importance of epigenetic regulation in the recovery of T cells following HSCT.
    MeSH term(s) Humans ; Cell Lineage ; Epigenesis, Genetic ; Hematopoietic Stem Cell Transplantation ; Graft vs Host Disease ; Leukemia
    Language English
    Publishing date 2023-01-23
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1459903-x
    ISSN 1521-7035 ; 1521-6616
    ISSN (online) 1521-7035
    ISSN 1521-6616
    DOI 10.1016/j.clim.2023.109245
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  10. Article ; Online: Thiotepa-Based Regimens Are Valid Alternatives to Total Body Irradiation-Based Reduced-Intensity Conditioning Regimens in Patients with Acute Lymphoblastic Leukemia: A Retrospective Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

    Battipaglia, Giorgia / Labopin, Myriam / Mielke, Stephan / Ruggeri, Annalisa / Nur Ozkurt, Zubeyde / Bourhis, Jean Henri / Rabitsch, Werner / Yakoub-Agha, Ibrahim / Grillo, Giovanni / Sanz, Jaime / Arcese, William / Novis, Yana / Fegueux, Nathalie / Spyridonidis, Alexandros / Giebel, Sebastian / Nagler, Arnon / Ciceri, Fabio / Mohty, Mohamad

    Transplantation and cellular therapy

    2023  Volume 30, Issue 1, Page(s) 95.e1–95.e10

    Abstract: Total body irradiation (TBI) at myeloablative doses is superior to chemotherapy-based regimens in young patients with acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, in elderly and ... ...

    Abstract Total body irradiation (TBI) at myeloablative doses is superior to chemotherapy-based regimens in young patients with acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, in elderly and unfit patients, in whom reduced-intensity conditioning (RIC) regimens are preferred, whether a TBI-based or a chemotherapy-based approach is better is unexplored. Thiotepa can be used as part of ALL conditioning regimens. The current study aimed to compare transplantation outcomes after RIC with TBI-based or thiotepa-based regimens in patients with ALL. The study cohort comprised patients aged ≥40 years undergoing allo-HSCT for ALL in first complete remission between 2000 and 2020 who received an RIC regimen containing either TBI (4 to 6 Gy) or thiotepa. We identified a total of 265 patients, including 117 who received a TBI-based RIC regimen and 148 who received a thiotepa-based RIC regimen. Univariate analysis revealed no significant differences in the following transplantation outcomes for TBI versus thiotepa: relapse, 23% versus 28% (P = .24); nonrelapse mortality, 20% versus 26% (P = .61); leukemia-free survival, 57% versus 46% (P = .12); overall survival, 67% versus 56% (P = .18); graft-versus-host disease (GVHD]/relapse-free survival, 45% versus 38% (P = .21); grade II-IV acute GVHD, 30% in both groups (P = .84); grade III-IV acute GVHD, 9% versus 10% (P = .89). The sole exception was the incidence of chronic GVHD, which was higher in the recipients of TBI-based regimens (43% versus 29%; P = .03). However, multivariate analysis revealed no differences in transplantation outcomes between the 2 groups. In patients aged ≥40 years receiving RIC, use of a thiotepa-based regimen may represent a valid alternative to TBI-based regimens, as no differences were observed in the main transplantation outcomes.
    MeSH term(s) Aged ; Humans ; Thiotepa/therapeutic use ; Retrospective Studies ; Whole-Body Irradiation/adverse effects ; Bone Marrow ; Leukemia, Myeloid, Acute/therapy ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy ; Acute Disease ; Graft vs Host Disease/prevention & control
    Chemical Substances Thiotepa (905Z5W3GKH)
    Language English
    Publishing date 2023-10-08
    Publishing country United States
    Document type Journal Article
    ZDB-ID 3062231-1
    ISSN 2666-6367
    ISSN (online) 2666-6367
    DOI 10.1016/j.jtct.2023.09.028
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