Elie Haddad's research while affiliated with Centre hospitalier universitaire de Sherbrooke and other places
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Publications (318)
Objectives
Inborn errors of immunity (IEI) are a heterogeneous group of genetic diseases that impact normal immune development and function. Individual IEI are rare, but collectively, can represent an important health burden. Little is known about the types of IEI seen in Canadian First Nations (FN) and Inuit populations. We sought to understand th...
Background
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the standard of care for chemotherapy-refractory leukemia patients, but cure rates are still dismal. To prevent leukemia relapse following HSCT, we aim to improve the early graft-versus-leukemia effect mediated by natural killer (NK) cells. Our approach is based on the ado...
Background
Triokinase and FMN cyclase (TKFC) is a bifunctional enzyme involved in fructose metabolism. Triokinase catalyses the phosphorylation of fructose-derived glyceraldehyde (GA) and exogenous dihydroxyacetone (DHA), while FMN cyclase generates cyclic FMN. TKFC regulates the antiviral immune response by interacting with IFIH1 (MDA5). Previousl...
We describe humans with rare biallelic loss-of-function PTCRA variants impairing pre–α T cell receptor (pre-TCRα) expression. Low circulating naive αβ T cell counts at birth persisted over time, with normal memory αβ and high γδ T cell counts. Their TCRα repertoire was biased, which suggests that noncanonical thymic differentiation pathways can res...
Objective:
To assess changes in juvenile idiopathic arthritis (JIA) treatments and outcomes in Canada, comparing a 2005-2010 and a 2017-2021 inception cohorts.
Methods:
Patients enrolled within three months of diagnosis in the Research in Arthritis in Canadian Children Emphasizing Outcomes (ReACCh-Out) and the Canadian Alliance of Pediatric Rheu...
Objectives
This study reports cases of systemic-onset juvenile idiopathic arthritis (sJIA) who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) at our center and reviews published outcomes of allo-HSCT in sJIA.
Methods
We present a case report of two patients with sJIA who underwent allo-HSCT at a tertiary pediatric hospita...
Neuroblastoma, the most common type of pediatric extracranial solid tumor, causes 10% of childhood cancer deaths. Despite intensive multimodal treatment, the outcomes of high-risk neuroblastoma remain poor. We urgently need to develop new therapies with safe long-term toxicity profiles for rapid testing in clinical trials. Drug repurposing is a pro...
Introduction/Background
Changes in relative virulence of SARS-CoV-2 lineages among children remain poorly understood, yet are important considerations for vaccination and health resource management. Further evidence is needed to assess the burden of severe paediatric COVID-19 due to the Omicron variant.
Objectives
In this study, we aimed to compar...
Background:
CGD is caused by defects in any of the 6 subunits forming the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase complex 2 (NOX2), leading to severely reduced or absent phagocyte-derived ROS production. Almost 50% of patients with chronic granulomatous disease (CGD) have IBD (CGD-IBD). While conventional IBD therapies can trea...
Introduction
Natural Killer (NK) cells hold the potential to shift cell therapy from a complex autologous option to a universal off-the-shelf one. Although NK cells have demonstrated efficacy and safety in the treatment of leukemia, the limited efficacy of NK cell-based immunotherapies against solid tumors still represents a major hurdle. In the im...
Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by life-threatening infections and inflammatory conditions. Hematopoietic cell transplantation (HCT) is the definitive treatment for CGD, but questions remain regarding patient selection and impact of active disease on transplant outcomes. We performed a multi-instituti...
Background:
Paediatric inflammatory multisystem syndrome (PIMS) is a rare condition temporally associated with SARS-CoV-2 infection. Using national surveillance data, we compare presenting features and outcomes among children hospitalized with PIMS by SARS-CoV-2 linkage, and identify risk factors for intensive care (ICU).
Methods:
Cases were rep...
Background:
Severe combined immunodeficiency (SCID) is fatal unless durable adaptive immunity is established, most commonly through allogeneic haematopoietic cell transplantation (HCT). The Primary Immune Deficiency Treatment Consortium (PIDTC) explored factors affecting the survival of individuals with SCID over almost four decades, focusing on t...
Background:
The nirmatrelvir/ritonavir has shown to reduce COVID-19 hospitalization and death before Omicron but updated real-world evidence studies are needed. This study aimed to assess whether nirmatrelvir/ritonavir reduces the risk of COVID-19-associated hospitalization among high-risk outpatients.
Methods:
This was a retrospective cohort st...
Background:
T lymphocytes exhibit numerous alterations in relapsing-remitting (RRMS), secondary progressive (SPMS), and primary progressive multiple sclerosis (PPMS). The NKG2D pathway has been involved in MS pathology. NKG2D is a co-activating receptor on subsets of CD4+ and most CD8+ T lymphocytes. The ligands of NKG2D are expressed at low level...
PurposeMetabolic detoxification with enzyme replacement therapy (ERT) promotes immune recovery in patients with adenosine deaminase (ADA)–deficient severe combined immunodeficiency (ADA-SCID). Elapegademase is a PEGylated recombinant bovine ADA ERT developed to replace the now-discontinued bovine-derived pegademase. This study was a 1-way crossover...
Introduction
The nirmatrelvir/ritonavir (PAXLOVIDTM) is an antiviral blocking the replication of SARS-CoV-2. Early treatment with this antiviral has showed to reduce COVID-19 hospitalization and death in unvaccinated outpatients with mild-to-moderate COVID-19 and high risk of progression to severe disease with variants before Omicron. However, the...
Background:
ADAGEN®, a bovine-based enzyme replacement therapy (ERT) has been used to treat adenosine deaminase severe combined immune deficiency (ADA-SCID). In 2018, ADAGEN® was replaced by REVCOVI®, (elapegademase) a modified bovine recombinant protein.
Objective:
To determine the real-life long-term benefits of REVCOVI® in ADA- SCID.
Methods...
Humanization of mice with functional T cells currently relies on co-implantation of hematopoietic stem cells from fetal liver and autologous fetal thymic tissue (so-called BLT mouse model). Here, we show that NOD/SCID/IL2rγnull mice humanized with cord blood- derived CD34+ cells and implanted with allogeneic pediatric thymic tissues excised during...
Current practices vary widely regarding the immunological work-up and management of patients affected with defects in thymic development (DTD), which include chromosome 22q11.2 microdeletion syndrome (22q11.2del) and other causes of DiGeorge syndrome (DGS) and coloboma, heart defect, atresia choanae, retardation of growth and development, genital h...
Multisystem inflammatory syndrome in children (MIS-C) is a rare and severe condition that follows benign COVID-19. We report autosomal recessive deficiencies of OAS1 , OAS2 , or RNASEL in five unrelated children with MIS-C. The cytosolic dsRNA-sensing OAS1 and OAS2 generate 2′-5′-linked oligoadenylates (2-5A) that activate the ssRNA-degrading RNase...
Neutralizing antibodies (NAbs) hold great promise for clinical interventions against SARS-CoV-2 variants of concern (VOCs). Understanding NAb epitope-dependent antiviral mechanisms is crucial for developing vaccines and therapeutics against VOCs. Here we characterized two potent NAbs, EH3 and EH8, isolated from an unvaccinated pediatric patient wit...
Severe combined immunodeficiency (SCID) results from defects in the differentiation of hematopoietic stem cells into mature T lymphocytes, with additional lymphoid lineages affected in particular genotypes. In 2014, the Primary Immune Deficiency Treatment Consortium published criteria for diagnosing SCID, which are now revised to incorporate contem...
Background
Shearer et al in 2014 articulated well-defined criteria for the diagnosis and classification of severe combined immunodeficiency (SCID) as part of the Primary Immune Deficiency Treatment Consortium’s (PIDTC’s) prospective and retrospective studies of SCID.
Objective
Because of the advent of newborn screening for SCID and expanded availa...
Acute lymphoblastic leukemia (ALL) is the most frequent cancer diagnosed in children. Despite the great progress achieved over the last 40 years, with cure rates now exceeding 85%, refractory or relapsed ALL still exhibit a dismal prognosis. This poor outcome reflects the lack of treatment options specifically targeting relapsed or refractory ALL....
Machine learning risk prediction of mortality for patients undergoing surgery with perioperative SARS-CoV-2: the COVIDSurg mortality score
Macrophages populate the embryo early in gestation but their role in the developmental process remains largely unknown. In particular, specification and function of macrophages in intestinal development remain unexplored. To study this event in human developmental context, we derived and combined human intestinal organoid and macrophages from pluri...
Background
Interleukin-27 (IL-27) can trigger both pro- and anti-inflammatory responses. This cytokine is elevated in the central nervous system (CNS) of multiple sclerosis (MS) patients, but how it influences neuroinflammatory processes remains unclear. As astrocytes express the receptor for IL-27, we sought to determine how these glial cells resp...
Direct comparisons of pediatric hospitalizations for acute COVID-19 and multisystem inflammatory syndrome in children (MIS-C) can inform health system planning. While there were more hospitalizations and deaths from acute COVID-19 amongst Canadian children between March 2020-May 2021, MIS-C cases were more severe, requiring more intensive care and...
Background
Severe combined immunodeficiency (SCID) comprises rare inherited disorders of immunity that require definitive treatment through hematopoietic cell transplantation (HCT) or gene therapy for survival. Despite successes of allogeneic HCT, many SCID patients experience incomplete immune reconstitution, persistent T-cell lymphopenia, and poo...
Granulocyte transfusions are sometimes used as adjunctive therapy for the treatment of infection in patients with chronic granulomatous disease (CGD). However, granulocyte transfusions can be associated with a high rate of alloimmunization, and their role in CGD patients undergoing hematopoietic cell transplantation (HCT) or gene therapy (GT) is un...
Objectives
To 1) validate the juvenile idiopathic arthritis (JIA) parent global assessment (parent global) as a health-related quality of life (HRQoL) instrument, 2) evaluate measurement properties of accepted HRQoL measures relative to those of the parent global, and 3) assess causal pathways determining parent global scores.
Methods
Data from th...
Adenosine deaminase (ADA) deficiency causes ~13% of cases of severe combined immune deficiency (SCID). Treatments include enzyme replacement therapy (ERT), hematopoietic cell transplant (HCT), and gene therapy (GT). We evaluated 131 ADA-SCID patients diagnosed between 1982-2017 who were enrolled in the Primary Immune Deficiency Treatment Consortium...
Generating humanized mice with fully functional T cells currently relies on co-implantation of hematopoietic stem cells from fetal liver and autologous fetal thymic tissue (BLT mouse). However, access to such tissues has ethical and logistical challenges. Herein, we show that NOD/SCID/IL2rγ null mice humanized with cord blood-derived CD34 ⁺ cells a...
Background
Paediatric inflammatory multisystem syndrome (PIMS) is a rare but serious condition temporally associated with SARS-CoV-2 infection. Using the Canadian Paediatric Surveillance Program (CPSP), a national surveillance system, we aimed to 1) study the impact of SARS-CoV-2 linkage on clinical and laboratory characteristics, and outcomes in h...
Introduction:
While 18F-FDG PET/CT pediatrics applications have increased in number and indications, few studies have addressed normal maximum standardized uptake values (SUVmax) of referral organs in children. The purpose of this study is to assess these in a cohort of pediatric patients.
Material and methods:
285 18F-FDG PET/CT scans in 229 pa...
Acute lymphoblastic leukemia (ALL) is the most frequent cancer diagnosed in children. Despite the great progress achieved over the last 40 years, with cure rates now exceeding 85%, refractory or relapsed ALL still exhibit a dismal prognosis. This poor outcome reflects the lack of treatment options specifically targeting relapsed or refractory ALL....
Modeling the tumor-immune cell interactions in humanized mice is complex and limits drug development. Here, we generated easily accessible tumor models by transforming either primary skin fibroblasts or induced pluripotent stem cell-derived cell lines injected in immune-deficient mice reconstituted with human autologous immune cells. Our results sh...
Congenital athymia can present with severe T cell lymphopenia (TCL) in the newborn period, which can be detected by decreased T cell receptor excision circles (TRECs) on newborn screening (NBS). The most common thymic stromal defect causing selective TCL is 22q11.2 deletion syndrome (22q11.2DS). T-box transcription factor 1 (TBX1), present on chrom...
Background and Objectives
We posit the involvement of the natural killer group 2D (NKG2D) pathway in multiple sclerosis (MS) pathology via the presence of specific NKG2D ligands (NKG2DLs). We aim to evaluate the expression of NKG2DLs in the CNS and CSF of patients with MS and to identify cellular stressors inducing the expression of UL16-binding pr...
SARS-CoV-2 has been associated with an increased rate of venous thromboembolism in critically ill patients.Since surgical patients are already at higher risk of venous thromboembolism than general populations, thisstudy aimed to determine if patients with peri-operative or prior SARS-CoV-2 were at further increased risk ofvenous thromboembolism. We...
There is little evidence around the potentially protective role of previous Bacillus Calmette-Guerin (BCG) vaccination on postoperative mortality in patients with perioperative SARS-CoV-2 vaccination. Prior BCG vaccination did not protect SARS-CoV-2 infected patients against postoperative pulmonary complications and 30-day mortality.
The efficacy of convalescent plasma for coronavirus disease 2019 (COVID-19) is unclear. Although most randomized controlled trials have shown negative results, uncontrolled studies have suggested that the antibody content could influence patient outcomes. We conducted an open-label, randomized controlled trial of convalescent plasma for adults with...
Umbilical cord blood transplantation (UCBT) has been used to treat malignant and non-malignant diseases. UCBT offers the advantages of easy procurement and acceptable partial HLA mismatches, but also shows delayed hematopoietic and immunological recoveries. We postulated that an intrabone (IB) infusion of cord blood could provide a faster short- an...
To support the global restart of elective surgery, data from an international prospective cohort study of 8492 patients (69 countries) was analysed using artificial intelligence (machine learning techniques) to develop a predictive score for mortality in surgical patients with SARS-CoV-2. We found that patient rather than operation factors were the...
Multisystem inflammatory syndrome in children (MIS-C) emerged in April 2020 in communities with high COVID-19 rates. This new condition is heterogenous but resembles Kawasaki disease (KD), a well-known but poorly understood and clinically heterogenous pediatric inflammatory condition for which weak associations have been found with a myriad of vira...
To fully perform their functions, T lymphocytes migrate within organs’ parenchyma and interact with local cells. Infiltration of T lymphocytes within the central nervous system (CNS) is associated with numerous neurodegenerative disorders. Nevertheless, how these immune cells communicate and respond to neural cells remains unresolved. To investigat...
Background:
Rituximab (RTX) is a B-cell depleting agent used in B-cell malignancies and autoimmune diseases. A subset of adult patients may develop prolonged and symptomatic hypogammaglobulinemia following RTX. However, this phenomenon has not been well delineated in the pediatric population.
Objective:
To determine the prevalence, risk factors...
Modeling the tumor-immune cell interactions in humanized mice is complex and limits drug development. Here, we generated easily accessible tumor models by transforming either primary skin fibroblasts or iPSC-derived cell lines injected in immune-deficient mice reconstituted with human autologous immune cells. Our results showed that either fibrobla...
Studies in murine models show that subthreshold T cell receptor (TCR) interactions with self‐peptide are required for thymic development and peripheral survival of naïve T cells. Recently, differences in the strength of tonic TCR interactions with self‐peptide, as read‐out by cell surface levels of CD5, were associated with distinct effector potent...
Peri-operative SARS-CoV-2 infection increases postoperative mortality. The aim of this study was to determine the optimal duration of planned delay before surgery in patients who have had SARS-CoV-2 infection. This international, multicentre, prospective cohort study included patients undergoing elective or emergency surgery during October 2020. Su...
A Correction to this paper has been published: https://doi.org/10.1007/s10875-020-00917-0
Purpose:
The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children with severe combined immunodeficiency (SCID) in a prospective natural history study of hematopoietic stem cell transplant (HSCT) outcomes over the last decade. Despite newborn screening (NBS) for SCID, infections occurred prior to HSCT. This study's objectives we...
Purpose
The Health state descriptive system includes standardized self-administered instruments for measuring Health-Related Quality of Life (HRQoL) respectively among adolescents, and children. The objectives of the current study were: (1) to translate and adapt the pediatric-adolescent version 16D and 17D from English into French (Canada), (2) to...
Introduction
Les mutations du Tetratricopeptide Repeat Domain 7A (TTC7A) causent un syndrome autosomique récessif combinant des atteintes digestives (atrésies intestinales multiples (AIM) et maladies inflammatoires de l’intestin) et une immunodéficience (ID). Des atteintes cutanées (ichtyose, alopécie, lésions psoriasiformes, onychodystrophies et k...
Cancer immunotherapy using genetically modified immune cells such as those expressing chimeric antigen receptors has shown dramatic outcomes in patients with refractory and relapsed malignancies. Natural killer (NK) cells as a member of the innate immune system, possessing both anticancer (cytotoxic) and proinflammatory (cytokine) responses to canc...
Reservoirs of HIV during ART are the primary reasons why HIV/AIDS remains an incurable disease. Indeed, HIV remains latent and unreachable by antiretrovirals in cellular and anatomical sanctuaries, preventing its eradication. The lungs have received very little attention compared to other anatomical reservoirs despite being immunological effector s...
The original version of this article unfortunately contained the missing author, Caridad Martinez. The authors would like to correct the list. We apologize for any inconvenience that this may have caused. The correct author list is shown above.
Targeted immunotherapy has improved the outcome of patients with high-risk neuroblastoma (NB). However, immune escape of tumor cells still occurs and about 40% of NB patients relapse and die from their disease. We previously showed that natural killer (NK) cell stimulation by Toll-like receptor (TLR)-activated plasmacytoid dendritic cells (pDC) inc...
It is still unclear if immune responses will compromise the large‐scale utilization of human induced pluripotent stem cells (hiPSCs)‐derived cell therapies. To answer this question, we used humanized mouse models generated by the adoptive transfer of peripheral blood mononuclear cells or the cotransplantation of hematopoietic stem cells and human t...
Isolated neuroinflammatory disease has been described in case reports of familial hemophagocytic lymphohistiocytosis (FHL), but the clinical spectrum of disease manifestations, response to therapy and prognosis remain poorly defined. We combined an international survey with a literature search to identify FHL patients with (i) initial presentation...
Key Points
3D organoid T-cell differentiation from a few hundred peripheral blood CD34+ cells was successfully achieved. 3D organoid T-cell differentiation could help physicians distinguish intrinsic from extrinsic defects underlying a clinical SCID phenotype.
Background & Aim
Acute liver failure (ALF) consists in a dramatic loss of liver functions resulting in a survival rate <50%. There is an urgent need for new therapies capable of replacing liver functions in patients with ALF. We developed a human induced pluripotent stem cell (iPSC)-derived Encapsulated Liver Tissue (ELT) capable of consistently pe...
Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outc...
Background
With >36 years of data collected, PIDTC prospective (6901) and retrospective (6902) natural history studies provide an unprecedented opportunity to study hematopoietic cell transplantation (HCT) outcomes for SCID over time.
Methods
Patients in this 6901/6902 analysis met PIDTC diagnostic criteria for SCID and underwent HCT. Categorial v...
Citations
... Helios deficiency, caused by a monoallelic truncating variant of IKZF2, a gene encoding a protein mostly expressed in T reg cells, has also been associated with autoantibodies against type I IFNs 97 . In one case, a patient with inherited complete pre-TCRα deficiency who had recurrent herpetic infections, was positive for autoantibodies that neutralize I IFNs 98 . Overall, a breakdown of central T cell tolerance, via mTECs and/or in a T cell-intrinsic manner, can drive the production of autoantibodies against type I IFNs. ...
Reference: The ouroboros of autoimmunity
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... Typically, these immune cells are blood-derived and are subsequently mixed with the organoid [97]. However, differentiating the immune cells from hiPSCs and integrating them into the 3D model would offer greater control over their numbers and types, and potentially even allow them to be isogenic [98,99]. Another approach could involve co-differentiating immune cells during cardioid differentiation, as was recently demonstrated with intestinal organoids [100], although challenges will remain in controlling the type and proportion of immune cells per cardioid. ...
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... The discovery of disulfiram, originally used to treat alcohol abuse, has been found to also have selective antineoplastic properties. This has been confirmed through genome-wide gene expression studies (Beaudry et al. 2023). ...
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... Higher biological age and lower physical fitness were significantly associated with increased Dorea abundance (44). Observational study results have shown a significant increase in Salmonella and Haemophilus in older individuals (45,46). Coprococcus 1 and Ruminococcus were found to have the strongest association with age-related phenotypes (47). ...
... Therefore, HSCT is an attractive option even for patients with inflammatory bowel disease as their sole CGD morbidity. [74][75][76][77] The liver is another part of the GI tract that can be affected by dysregulated inflammation, with complications including non-cirrhotic portal hypertension, nodular regenerative hyperplasia, and hepatosplenomegaly resulting in sequestration and thrombocytopenia. 13,78 Approximately one quarter of patients with CGD exhibit signs of pulmonary inflammatory complications. ...
... Nonetheless, this ideal scenario is the least frequent, unless a newborn screening program is conducted, or family history is present. [9,16] It highlights the importance of identifying other HSCT factors that might enhance patient survival, such as age, infection status at the time of HSCT, phenotype, genotype, and the type of available donor. This evaluation can be helpful to offer the best HSCT scenario for each patient. ...
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... Recent case examples of the inclusion of SCID in NBS programmes worldwide, discussed during the session, underscored the importance of healthcare system preparedness for the inclusion of a new disease and the need for timely evaluation to ensure that the programme brings more benefit than harm [11]. For SCID, it has been shown that earlier diagnosis and consequently earlier therapeutic intervention through allogenic stem cell transplantation or autologous gene therapy have significantly improved the overall survival of treated SCID babies [12,13]. Indeed, the importance of working towards equity and innovation in NBS has been highlighted by several initiatives, including the Screen4Rare coalition and the recently launched European Reference Network (ERN) Expert Platform for Newborn Screening, and was a key action point identified during IPOPI's 2022 Global Multi-Stakeholder's Summit [14]. ...
... They reported an increased protective activity of N/r, with an 81% risk reduction among incompletely vaccinated individuals and patients who had received their most recent vaccine dose more than 20 weeks prior (19). In a population-based cohort study conducted in Quebec, Canada, a 96% reduction in the hospitalisation rate was reported among incompletely vaccinated outpatients (22). However, in this cohort, no benefit was observed among the vaccinated population, excluding severely immunocompromised patients and high-risk patients aged 70 years or older who had received their last vaccine dose more than 6 months prior. ...
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... In 2019, the use of ADAGEN was discontinued, and Revcovi became the only available form of ERT for ADA deficiency. The clinical trials that led to FDA approval of Revcovi primarily included adults and children who were previously treated for many years with ADAGEN [5]. There are limited data on the use of Revcovi to treat ERT-naïve infants with ADA-SCID [6]. ...
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... Leg PedThy mice were produced by surgical engraftment of human pediatric thymus from a single donor (Donor 1) into the quadriceps muscle followed by HSC injection (Table 1), as previously described 9 . To compare the effects of myeloablation on immune reconstitution, mice were myeloablated with sublethal irradiation (n = 3) or busulfan injection (n = 6) or were not myeloablated (n = 3) and followed for up to 24 weeks post-engraftment. ...
