Immunogenetics in sickle cell disease. Karina Tozatto Maio 2018 USP-USPC Workshop on Hematology and Bone Marrow Transplant 11/05/2018
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1 Immunogenetics in sickle cell disease Karina Tozatto Maio 2018 USP-USPC Workshop on Hematology and Bone Marrow Transplant 11/05/2018
2 Sickle cell disease Monogenic disorder Extremely variable phenotype Asymptomatic x transplanted patients
3 Kato GJ et al. Nat Rev Dis Primer, 2018
4 Kato GJ et al. Nat Rev Dis Primer, 2018
5 Phenotype severity x genetics What causes phenotype heterogeneity? Can inflammatory polymorphisms be involved? Piel et al NEJM 2017
6
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8 HCST and SCD Outcomes after HSCT for SCD. (A) Unadjusted chronic GVHD, according to age; and (B) OS, according to stem cell source. 5-y OS 93%, 5-y EFS 91% (n=1000, MRD) CI GvHD 14%, < usually described why? Lack of HLA identical stem cell donor Eliane Gluckman et al. Blood 2017;129: by American Society of Hematology
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10 SCD and HLA African/Asian origin Migration Mixed haplotypes Ethnic minorities Lack of epidemiological studies Lack of genetic studies Lack of suitable stem cell donors
11 Objectives HLA and non HLA functionally relevant inflammatory polymorphisms Correlate with phenotypic variability Correlate with HSCT outcomes HLA: improve stem cell donor search Steady state inflammatory status of SCD: trigger
12 Endpoints Primary endpoint: - To describe allele/genotype/haplotype frequencies of HLA, NK cell receptors, cytotoxic T lymphocyte associated protein 4 (CTLA-4) and Toll-like receptor polymorphisms in SCD patients Secondary endpoints: - To compare allele/genotype/haplotype frequencies in SCD clinical subgroups - Transplanted patients: to analyze the impact of different allele/genotype/haplotype frequencies on HSCT outcomes - HLA: to calculate the probability of finding an unrelated donor in international donor registries
13 Methods (I) Two cohorts Cohort 1 HLA data available Cohort 2 DNA available Different methods Same endpoints (association among inflammatory polymorphisms and clinical outcomes)
14 Methods (II) Cohort 1: Eurocord/EBMT transplanted patients from EBMT centers - HLA and clinical information database
15 Methods (III) Cohort 1: HLA allele group/haplotype level Clinical manifestations presence of infections CNS vasculopathy alloimmunization transfusion reactions HSCT outcomes Describe HLA allele group/haplotype frequencies Calculate probability of finding a MUD in donor registries
16 Methods (IV) Cohort 2: 524 transplanted and non transplanted SCD patients N=56 N=217 N=107 N=144 CHU Dakar, CHU Tenon, HC-FMUSP, HC-FMRP-USP, Senegal France Brazil Brazil Available DNA and clinical data
17 Methods (V) Cohort 2: SNPs in inflammatory genes Clinical manifestations presence of infections CNS vasculopathy alloimmunization transfusion reactions HSCT outcomes
18 Statistical considerations Polymorphism analysis: - logistic regression correlation with clinical variables HSCT results: - OS/EFS: Kaplan-Meyer - GvHD/RI: CI curves - UVA: p<0.15 or clinical relevance MVA (Cox regression)
19 Partial results - Demography
20 Tenon + Senegal (n=250, non transplanted patients) Ribeirão Preto (n=151, transplanted n=32) EBMT centers (n=727, all transplanted) Median age 33 (8-69) 24 (7-59) 9 (0-39) Female gender 57% 55% 47% SS + SB SC 83% 100% 99% 17% 0 1% HU therapy 44% 81% 58% Previous transfusions 66% 90% 94% Chronic complications cholelithiasis cholelithiasis liver disease osteonecrosis sickle nephropathy osteonecrosis retinopathy low bone mass sickle nephropathy Acute complications VOC VOC VOC ACS bacterial infections ACS bacterial infections ACS stroke Main HSCT indications NA cerebral vasculopathy cerebral vasculopathy VOC VOC
21 Partial results HSCT according to age
22 N= 727 SCD patients HLA identical sibling donor Median follow-up: 46 months OUTCOME general group yo group yo group 3 >15 yo P-value (Log-Rank) 3y-OS 95% +1 99% % % + 3 < y-EFS 92% % % % day engraftment 97% % % % day agvhd 15%+ 1 9% % % y cgvhd 12%+1 9%+2 12%+2 20% Cappelli et al. ASH 2017
23 Partial results TLR-2 polymorphism and bacterial infections in SCD patients
24 Rationale Infection leading cause of mortality in SCD 160 SCD patients from France and Senegal with adequate immunization and penicillin prophylaxis Occurrence of bacterial infections x SNPs in inflammatory genes Tozatto-Maio et al. EHA 2017
25 Toll like receptors TLR: transmembrane proteins Pattern recognition receptors TLR-2: pivotal receptor Innate immune system Rakoff-Nahoum Nat Rev Cancer 2009
26 Results (I) 76 infected patients x 84 non infected patients No differences in gender, age and SCD genotype distribution Most common etiological agents: encapsulated bacteria (S. pneumoniae, E. coli, Klebsiella spp), Mycobacterium tuberculosis Tozatto-Maio et al. EHA 2017
27 Results (II) SNP genotype infected (%) x cp-value OR 95%CI others (%) infected (n=76) vs rs T/A 68 (45%) x < not infected (n=84) 164 (98%) encapsulated (n=35) rs T/A 18 (51%) x < vs others (n=107) 91 (85%) TLR-2 rs genotype T/A ideal inflammatory modulation wild type (T/T): weak response mutant (A/A): overresponse Tozatto-Maio et al. EHA 2017
28 Annalisa Ruggeri, MD Franco Locatelli, MD Eurocord Monacord 2018 Vanderson Rocha, MD PhD Barbara Cappelli Lauro, MD Chantal Kenzey, CRA Hanadi Rafii Elayoubi, MD Eliane Gluckman, MD FRCP Fernanda Volt, MT Graziana Scigliuolo, PharmD Agnès Devergie, MD Hiromi Hayashi, MD Karina Tozatto Maio, MD Fabienne Guenoun, AP
29 Acknowledgments Eurocord/Monacord Eliane Gluckman Annalisa Ruggeri Vanderson Rocha Fernanda Volt Chantal Kenzey Barbara Cappelli Hanadi Elayoubi Hiromi Hayashi Graziana Scigliuolo Fabienne Guenoun CHU Tenon Robert Girot Laurent Benzerara CHU Dakar Ibrahim Diagne Indou Deme Ly Department of Hematology HC FMUSP Vanderson Rocha Carla L. Dinardo Guilherme Fonseca Sandra Gualandro Department of Hematology HC FMRP USP Belinda Pinto Simões Ana Cristina Silva Pinto Luciana Jarduli Thalita Costa Simone Kashima INSERM U942 Ryad Tamouza Christina Mariaselvam Celine Manier INSERM U1160 Itaua Leston Araujo Emmanuel Clave
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