Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT

Neven, Bénédicte; Ferrua, Francesca

doi:10.3389/fped.2019.00552

Cited by 16 publications

(10 citation statements)

References 75 publications

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“…The impact of comorbidity on transplant outcome is also reflected in the observation that in various single disease studies younger age at transplant as well as shorter time from diagnosis to transplant, are correlated with better overall and event-free survival [3,7]. In order to address the fact that the most appropriate conditioning regimen may also be disease specific, a recently published review series on behalf of IEWP covering HSCT in different CID disease identities provides more disease-specific recommendations [8,67,69,71]. Specific considerations.…”

Section: Combined Immunodeficienciesmentioning

confidence: 99%

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

Lankester

Albert²,

Booth

et al. 2021

Bone Marrow Transplant

126

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Section: Combined Immunodeficienciesmentioning

confidence: 99%

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

Lankester

Albert²,

Booth

et al. 2021

Bone Marrow Transplant

126

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“…[16][17][18][19][20] Other microbiologic factors associated with post-HCT lung injury include pre-HCT cytomegalovirus seropositivity and history of pulmonary bacterial or invasive fungal infection. [21][22][23][24][25][26] Finally, host factors, including elevations in baseline inflammation and tissue injury, may predispose to post-HCT lung injury, although these data are sparse. [27][28][29][30][31] To further elucidate pre-HCT biological factors associated with post-HCT lung injury, we applied advances in metatranscriptomic sequencing to a unique cohort of pediatric HCT candidates who underwent routine pre-HCT bronchoalveolar lavage (BAL).…”

Section: Introductionmentioning

confidence: 99%

The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury

Zinter

Lindemans

Versluys

et al. 2021

Blood

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Lung injury after pediatric allogeneic hematopoietic cell transplantation (HCT) is a common and disastrous complication that threatens long-term survival. In order to develop strategies to prevent lung injury, novel tools are first needed to comprehensively assess lung health in HCT candidates. Therefore, this study analyzed biospecimens from 181 pediatric HCT candidates who underwent routine pre-HCT bronchoalveolar lavage (BAL) at the University Medical Center in Utrecht, the Netherlands between 2005-2016. BAL fluid underwent metatranscriptomic sequencing of microbial and human RNA and unsupervised clustering and generalized linear models were used to associate microbiome-gene expression data with the development of post-HCT lung injury. Microbe-gene correlations were validated using a geographically distinct cohort of 18 pediatric HCT candidates. The cumulative incidence of post-HCT lung injury varied significantly according to four pre-HCT pulmonary metatranscriptome clusters, with the highest incidence observed in children with pre-HCT viral enrichment and innate immune activation and in children with profound microbial depletion and concomitant NK/T-cell activation (p<0.001). In contrast, children with pre-HCT pulmonary metatranscriptomes containing diverse oropharyngeal taxa and lacking inflammation rarely developed post-HCT lung injury. In addition, activation of epithelial-epidermal differentiation, mucous production, and cellular adhesion were associated with fatal post-HCT lung injury. In a separate validation cohort, associations between pulmonary respiratory viral load, oropharyngeal taxa, and pulmonary gene expression were recapitulated; the association with post-HCT lung injury needs to be validated in an independent cohort. This analysis suggests that assessment of the pre-HCT BAL fluid may identify high-risk pediatric HCT candidates who may benefit from pathobiology-targeted interventions.

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“…The method is a potentially curative therapeutic approach and is mostly used among patients with hematologic malignancies and myelodysplastic syndromes. It is also introduced in the solid tumors treatment schemes, as well as bone marrow failure syndromes, primary immunodeficiencies, and inborn errors of metabolism [2][3][4]. Depending on the treatment protocol, a patient can be a self-donor (autologous transplantation) or another unrelated person can be a donor for the patient (allogeneic transplantation) [1][2][3].…”

Section: Introductionmentioning

confidence: 99%

“…It is also introduced in the solid tumors treatment schemes, as well as bone marrow failure syndromes, primary immunodeficiencies, and inborn errors of metabolism [2][3][4]. Depending on the treatment protocol, a patient can be a self-donor (autologous transplantation) or another unrelated person can be a donor for the patient (allogeneic transplantation) [1][2][3]. The procedure involves hematopoietic stem cell collection from peripheral blood after mobilization of stem cells with granulocyte-colony-stimulating factor from bone marrow, bone marrow collection, or cord blood collection from the donor.…”

Section: Introductionmentioning

confidence: 99%

Lipid Complications after Hematopoietic Stem Cell Transplantation (HSCT) in Pediatric Patients

et al. 2020

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HSCT (hematopoietic stem cell transplantation) is a widely applied method of treatment of pediatric patients with leukemia and other bone marrow-associated disorders. Metabolic disturbances can appear as procedure side effects. This study aimed to report incidence of lipid and thyroid disorders and time of their onset in pediatric patients after HSCT. There were 198 pediatric patients (123 males) aged 0.5–20 years who were subjected to HSCT. Patients were mostly diagnosed with Acute Leukemia (n = 190). The analysis of lipids, thyroid hormones, and thyroid antibodies levels comprised one month before the HSCT to last follow up visit between 2016 and 2019 (median 3.8 ± 1.8 years after HSCT). In males, the triglycerides levels increased over two times in the course of HSCT in both patients with initially low and elevated HDL (high-density lipoprotein) levels. Most of the lipid disorders occurred in six months after HSCT. Patients treated with L-thyroxine exhibited decreased LDL (low-density lipoprotein) levels. HDL remained at a lower level in males. Thyroid hormone abnormalities were evenly distributed in time until 4 years after HSCT. Patients require long term follow up including lipid metabolism and thyroid function analysis. HSCT survivors demand introduction of polyunsaturated fatty acids into the diet to reduce risk of developing the lipid complications.

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Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT

Cited by 16 publications

References 75 publications

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

The pulmonary metatranscriptome prior to pediatric HCT identifies post-HCT lung injury

Lipid Complications after Hematopoietic Stem Cell Transplantation (HSCT) in Pediatric Patients

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