Background and aims: The markers of renal damage defining subclinical AKI are not widely used in children undergoing allogeneic hematopoietic stem cell transplantation (alloHSCT). The aim of the study was to evaluate serum and urinary clusterin as indices of kidney injury after alloHSCT in relation to damage (kidney injury molecule (KIM)-1) and functional (cystatin C) markers. Material and methods: Serum and urinary clusterin, KIM-1 and cystatin C concentrations were assessed by ELISA in 27 children before alloHSCT, 24 h, 1, 2, 3 and 4 weeks after alloHSCT and in controls. Results: All parameters were significantly higher in HSCT patients compared to controls even before the transplantation. The serum concentrations increased after HSCT and this rising trend was kept until the third (clusterin) or 4th (KIM-1, cystatin C) week. Urinary clusterin and KIM-1 were elevated until the third week and then decreased yet remained higher than before HSCT. Urinary cystatin C has risen from the second week after HSCT and decreased after the third week but was still higher than before alloHSCT. Conclusions: The features of kidney injury are present even before alloHSCT. Clusterin seems useful in the assessment of subclinical AKI and may become a new early marker of sublethal kidney injury in children.
Background. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a lifesaving procedure in malignant and nonmalignant diseases. However, it is associated with a considerable risk of graft-versus-host disease (GvHD). Steroids are a first-line therapy for acute GvHD (aGvHD), but there is no standard treatment for steroid-resistant (SR) gastrointestinal (GI) aGvHD, which has a poor prognosis. The anti-integrin antibody, vedolizumab, could help in controlling SR GI aGvHD symptoms by blocking lymphocyte extravasation and infiltration of the intestinal wall.
Objectives.To report the outcomes of 3 children with SR GI aGvHD after allo-HSCT, treated with vedolizumab as the last chance drug.
Materials and methods.The study included 3 patients aged from 8 to 10 years who underwent HSCT
IntroductionAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is a treatment method for a wide range of malignant and non-malignant diseases. Infants constitute a distinct patient group, especially due to their organ immaturity and differences in drug metabolism. The present paper aims to analyse the short- and long-term outcomes after allo-HSCT in infants.Material and methodsIn the study period, 67 patients under 12 months of age underwent allo-HSCT. This study is a retrospective analysis of patient medical records, in the form of paper and electronic documentation.ResultsThe probability of 5-year OS was 69% and 72% in patients with malignant and non-malignant diseases, respectively. The allo-HSCT from a matched donor was associated with improved OS in comparison to haploidentical donor (0.8 vs. 0.58%, p = 0.0425). The overall incidence of acute graft-vs.-host disease (aGVHD) was 59.3%, and grade III–IV aGVHD was diagnosed in 23% of patients. The 100-day non-relapse mortality (NRM) in the study cohort was 17.9%, while the 5-year NRM was 26.9%. Among the causes of NRM, infections occurred in 83.3% of patients, and aGVHD in 16.3% of individuals. Twenty-two children (32.8%) required hospitalization in the pediatric intensive care unit (PICU). The median length of PICU hospitalization was 6 days (range 1 to 12 days). Late sequelae diagnosed during post-transplant surveillance included ocular disorders in 26.8% of patients, cardiac complications in 4.4%, as well as endocrinopathy with short stature (<3rd percentile) in 37.2% and overt hypothyroidism in 35.4%. In the long-term perspective, 83.3% of survivors were able to attend a regular school.ConclusionsImprovements in unrelated donor availability, and better supportive care resulted in better outcomes. Management of infant allo-HSCT recipients requires the formation of multi-disciplinary specialist teams. In addition, the role of parental empowerment must be acknowledged; for example, in speech therapy and rehabilitation.
Krabbe disease (KD) is a lysosomal storage disorder, caused by a deficiency of galactocerebrosidase (GALC) or a mutation in the prosaposin gene, which leads to psychosine accumulation. Consequently, demyelination and degradation of the nervous system occur. Newborns with early infantile Krabbe disease usually present with poor feeding, irritability, hypertonicity, deafness and visual impairment. They usually die, unless transplanted with hematopoietic stem cells (HSC) during the 1st month of life. Patients with late form KD have heterogeneous symptoms, like ataxia or spastic paresis followed by slow progression. Therapeutic options are limited and hematopoietic stem cell transplantation (HSCT) remains the only life-saving method. In this report, we describe the first HSCT in late-infantile Krabbe disease in Poland. The post-transplant period was complicated by graft versus host disease (GvHD), veno-occlusive disease (VOD), mucositis and impairment of renal function. The 5-year follow-up was uneventful, confirming clinical efficacy of the HSCT.
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