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The 45Th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians – Oral Session

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The 45Th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians – Oral Session Bone Marrow Transplantation (2019) 54:16–141 https://doi.org/10.1038/s41409-019-0562-9 MEETING ABSTRACTS The 45th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians – Oral Session © Springer Nature Limited 2019 24–27 March 2019 ● Frankfurt, Germany Modified and published with permission from https://www.ebmt.org/annual-meeting Sponsorship Statement: Publication of this supplement is sponsored by the European Society for Blood and Marrow Transplantation. All content was reviewed and approved by the EBMT Committee, which held full responsibility for the abstract selections. Presenting author names are bold type in the contributor lists. 1234567890();,: 1234567890();,: Acute leukaemia Methods: Study aim was to evaluate the schedule of IST given in combination with PT-Cy as GVHD-prophylaxis O010 post-haplo for acute leukemia (AL) and reported to the The impact of the starting day of graft-versus-host ALWP/EBMT registry. Patients were divided into 3 groups: disease prophylaxis on haploidentical transplantation received cyclosporine A-mycofenolate-mofetil(CSA- with post-transplant cyclophosphamide: A retrospective +MMF) initiated at day+1 (group-1, n=124) or CSA study on behalf of acute leukaemia working party- +MMF both started at day+5 (group-2, n=170) and EBMT tacrolimus + MMF from day+5 (group-3, n=215). Transplants were performed from 2006-2017 and median Annalisa Ruggeri1, Myriam Labopin2, Johanna Tischer3, follow up is 21 months (range 11-36). PT-CY was given on Jean - Luis Diez Martin4, Benedetto Bruno5, Simona Day+3 and Day+5 in group-1 and on day+3 and day+4in Sica6, Luca Castagna7, Boris Afanasyev8, Antonin Vitek9, group-2 and 3. Montserrat Rovira10, Arnon Nagler11, Mohamad Mohty2 Results: Acute myeloid leukemia (AML) was the most common indication for haplo (76%) and approximately 1Ospedale Pediatrico Bambin Gesú, Roma, Italy, 2Saint 45% of patients were transplanted in CR1. There were some Antoine Hospital, Paris, France, 3BMT Unit, Muenchen, differences among groups: patients in group-1 were Germany, 4BMT Unit, Madrid, Spain, 5Le Molinette, younger (median age 46 years, p< 0.02) were transplanted Torino, Italy, 6Universita Cattolica, Roma, Italy, 7Humani- in more recent year (2015, p< 0.001), received more tas Clinical and Research Center Rozzano, Milano, Italy, frequently a regimen based on TBF (thiotepa, fludarabine 8BMT Unit, St Petersburg, Russian Federation, 9BMT Unit, and busulfan) (83%, p< 0.001) and bone marrow (BM) as Prague, Czech Republic, 10BMT Unit, Barcelona, Spain, source of stem cells (77%, p< 0.001), with no ATG (100%, 11Sheba Medical Center, Tel Aviv, Israel p< 0.001). Probability of OS at 2 years was 59%, 48% and 44%, for the 3 groups, respectively, p=0.15. Probability of Background: Post-transplant-Cyclophosphamide (PT-Cy) LFS and GRFS at 2 years were 52% and 46%, 43% and is being used successfully in the setting of unmanipulated 36%, 39% and 33%, for the 3 groups, respectively, (LFS haploidentical-transplant (haplo) as graft-versus-host-dis- p=0.05, GRFS p=0.01. Overall the cumulative incidence ease(GVHD) prophylaxis. However, the combined immu- (CI) of grade II-IV acute GVHD was 18%, 39% and 25%, nosuppressive compounds (IST) used as GVHD- for the 3 groups, respectively, p< 0.001, and the CI of prophylaxis and the post-transplant initiation day varies chronic GVHD was 23%, 21% and 25%; p=0.28. The CI of and is not fully standardized. relapse at 2 years was 26%, 37% and 35% (p=0.01) and The 45th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians. 17 non-relapse mortality (NRM) was 21%, 20% and 26% and investigate the interplay with NKG2D, a danger (p=0.34) for group-1, 2 and 3, respectively. Disease detector expressed by cytotoxic lymphocytes such as nat- recurrence, infections and GVHD were the most common ural killer (NK) cells that recognizes stress-induced ligands causes of death in the whole cohort.In multivariate analysis, (NKG2DL) of the MIC and ULBP protein families on the early start of MMF at day+1 in addition to PT-CY was AML cells. associated with reduced risk of chronic GVHD (HR 0.48, Methods: 175 de novo AML were stained with p=0.03) and improved GRFS (HR 0.63, p=0.02) while the antibodies against MICA, MICB and ULB2/5/6 or an effect on relapse was not confirmed. Stem cell source and NKG2D-Fc chimeric protein recognizing pan-NKG2DL. type of conditioning regimen did not influenced the NKG2DLpos and NKG2DLneg AML cells sorted from the outcomes in multivariate analysis.The timing of immuno- same patient were analysed in colony forming assays, suppression did not influenced NRM and survival. Diag- leukemogenesis assays in NSG mice, by RNAseq, gene nosis of ALL (HR 1.64, p=0.04), performance-status less expression arrays, qRT-PCR and targeted next generation than 90% (KPS90) (HR 1.72, p=0.001), were associated sequencing. AML cells co-cultured or not with NK cells with increased risk of NRM. Older age (HR 0.87, p=0.02), (control or anti-NKG2D pre-treated) were co-stained for and active disease (HR 3.58, p< 0.001) were associated with additional stem/immunological markers. PARP1 expression increased risk of relapse. KPS (OS, HR 0.59, p< 0.01; LFS, was analysed by qRT-PCR and immunoblot, and binding to HR 0.62, p< 0.001) and active disease (OS, HR 2.3, p< NKG2DL promoters by chromatin immunoprecipitation. 0.01; LFS, HR 2.43, p< 0.001) were independently PARP1 inhibition (PARPi) in AML cells was performed associated with reduced risk of OS and LFS. in vitro or in vivo using siRNAs or inhibitors (AG-14361, Conclusions: The time of starting of immunosuppression veliparib). drugs after haplo in combination with PT-Cy influences the Results: Heterogeneous NKG2DL expression was results of unmanipulated haploidentical transplant. The detected among leukemic cells of the same patient (Fig. early starting of CSA+MMF at day+1 decreases the risk of 1a). Interestingly, when compared to NKG2DLpos subpo- chronic GVHD, and improves GRFS. pulations, NKG2DLneg AML cells isolated from the same Disclosure: No disclosure patient showed immature morphology, enhanced in vitro clonogenicity (39±47 colonies vs. 1±4, p< 0.001, n=32 O011 AML patients) and selective abilities to initiate leukemia Human acute myeloid leukemia stem cells selectively (NKG2DLneg, 64/70, 91%; NKG2DLpos, 0/78, 0%; p< escape NKG2D-mediated NK cell control 0.00001, Fig. 1b, n=19 AML patients) and survive chemotherapy in NSG mice devoid of functional NK cells. Anna Paczulla1, Kathrin Rothfelder2,3, Simon Raffel4,5,6, In mice, NKG2DLneg AML cells generated both Martina Konantz1, Julia Steinbacher2,3, Hui Wang1, NKG2DLpos and NKG2DLneg progeny of which again only Marcelle Christine Ndoh Mbarga1, Thorsten Schäfer1, latter was able to induce leukemia in re-transplant assays. Daniela Dörfel3, Mattia Falcone4, Claudia Tandler2,3, Similar leukemia-specific mutations were detected in Jakob Passweg1, Pontus Lundberg1, Lothar Kanz3, Leticia NKG2DLneg compared to NKG2DLpos AML cells from Quintanilla-Martinez3, Alexander Steinle7, Andreas the same AML but published LSC (Fig. 1c), HSC and 17- Trumpp4,5, Helmut R. Salih2,3, Claudia Lengerke1 genes stemness score signatures were specifically enriched in NKG2DLneg subfractions. NKG2DLneg cells enriched for 1University and University Hospital Basel, Basel, Switzer- LSCs defined by alternative markers (CD34+, CD38-, land, 2German Cancer Consortium (DKTK), Partner Site GPR56+) but could identify cells with functional and Tuebingen, Heidelberg, Germany, 3University of Tuebin- molecular LSC activity also in CD34 non-expressing AML gen, Tuebingen, Germany, 4Deutsches Krebsforschungs- (n=11 analyzed patients). NKG2DL expression was zentrum (DKFZ), Heidelberg, Germany, 5Heidelberg repressed by PARP1 recruitment at NKG2DL promoters. Institute for Stem Cell Technology and Experimental PARP1 inhibition (PARPi) induced NKG2DL surface Medicine (HI-STEM gGmbH), Heidelberg, Germany, expression in LSCs and co-treatment with PARPi and NK 6University of Heidelberg, Heidelberg, Germany, 7Goethe cells (but not with either alone) suppressed leukemogenesis University Frankfurt, Frankfurt, Germany in patient derived xenograft (PDX) models (Fig. 2c) co- transplanted with NK cells. Low NKG2DL surface or high Background: Patients with acute myeloid leukaemia PARP1 mRNA expression associated with poor outcome in (AML) often achieve remission but subsequently die of AML patients. Furthermore, NKG2DLneg and CD34+ LSCs relapse driven by chemotherapy resistant leukemic stem showed reduced expression of other immune stimulatory cells (LSCs). Here we hypothesized that LSCs must also molecules (e.g. CD112, CD155, CD80, CD86) and different escape immunosurveillance to initiate and maintain cancer 18 expression of immune or inflammatory response gene transplantation (SCT) in Philadelphia chromosome positive signatures (GSEA). (Ph+) acute lymphoblastic leukemia (ALL) are scarce. Conclusions: These data indicate that LSCs escape NK Methods: We performed a retrospective, EBMT registry cell recognition by selectively suppressing the surface based analysis, including patients with documented use of expression of NKG2DL and other immunostimulatory 2nd or 3rd generation TKI given for persisting MRD, MR or molecules. Absence of NKG2DL can identify LSCs across HR after alloSCT in 2006-2016. Choice of TKI, efficacy, genetic AML subtypes (including CD34 negative AMLs). and toxicity of TKI and patient outcome were analysed. This LSC specific mechanism of immune evasion could be Results: 140 patients (female 58, male 82) were overcome by treatment with PARP1 inhibitors, which in identified, out of which 136 had also received a TKI conjunction with functional NK cells holds promise to (78% imatinib) before alloSCT. Median age at transplant eradicate LSCs and promote immune-mediated cure was 48 years (18-66), 81% were transplanted in first of AML. complete remission (CR1), 51% of the patients were in Disclosure: C.L.: Sanofi, Novartis, Otsuka (consultancy); molecular CR. Conditioning was myeloablative in 71% and Roche (research funding) reduced in 29%, donors were matched siblings (48%), unrelated (41%), haploidentical (6%) and cord blood (5%).
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