Dataset Information

Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide.

ABSTRACT:

Objective

We conducted an open-label, phase I study using autologous umbilical cord blood (UCB) infusion to ameliorate type 1 diabetes (T1D). Having previously reported on the first 15 patients reaching 1 year of follow-up, herein we report on the complete cohort after 2 years of follow-up.

Research design and methods

A total of 24 T1D patients (median age 5.1 years) received a single intravenous infusion of autologous UCB cells and underwent metabolic and immunologic assessments.

Results

No infusion-related adverse events were observed. β-Cell function declined after UCB infusion. Area under the curve C-peptide was 24.3% of baseline 1 year postinfusion (P < 0.001) and 2% of baseline 2 years after infusion (P < 0.001). Flow cytometry revealed increased regulatory T cells (Tregs) (P = 0.04) and naive Tregs (P = 0.001) 6 and 9 months after infusion, respectively.

Conclusions

Autologous UCB infusion in children with T1D is safe and induces changes in Treg frequency but fails to preserve C-peptide.

SUBMITTER: Haller MJ

PROVIDER: S-EPMC3220832 | biostudies-literature | 2011 Dec

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide.

Haller Michael J MJ Wasserfall Clive H CH Hulme Maigan A MA Cintron Miriam M Brusko Todd M TM McGrail Kieran M KM Sumrall Theresa M TM Wingard John R JR Theriaque Douglas W DW Shuster Jonathan J JJ Atkinson Mark A MA Schatz Desmond A DA

Diabetes care 20111019 12

<h4>Objective</h4>We conducted an open-label, phase I study using autologous umbilical cord blood (UCB) infusion to ameliorate type 1 diabetes (T1D). Having previously reported on the first 15 patients reaching 1 year of follow-up, herein we report on the complete cohort after 2 years of follow-up.<h4>Research design and methods</h4>A total of 24 T1D patients (median age 5.1 years) received a single intravenous infusion of autologous UCB cells and underwent metabolic and immunologic assessments. ...[more]

PMID: 22011412

Similar Datasets

Project description:BackgroundCurrently, the treatment of anemia in preterm infants is based on packed red blood cell (RBC) transfusions from adult donors. Oxygen (O2) is mainly transported to the tissues bound to hemoglobin (Hb). In extremely low gestational age neonates (ELGANs), fetal hemoglobin (HbF), which has a higher affinity for O2, represents up to 95% of circulating hemoglobin. During the first month of life, the majority of ELGANs will require an adult-donor RBC transfusion causing HbF levels to rapidly drop. HbA releases 50% more oxygen in peripheral tissues than HbF. Increased release of O2 in the retina is one of the main factors related to the development of retinopathy of prematurity (ROP). Collecting umbilical cord blood and using autologous umbilical cord whole blood (UCB) transfusions would contribute to maintaining physiological HbF concentrations in newborns and avoid oxygen-in-excess derived damage.MethodsThis is a randomized, double-blinded, multicenter clinical trial. ELGANs ≤28 weeks of gestational age will be randomized 1:1 to receive an autologous umbilical cord blood transfusion (intervention arm) or standard transfusion of packed RBC from an adult donor (control arm) to assess ROP development. Assuming a 50% reduction in ROP incidence, 134 patients (67 per group) will be recruited. When blood transfusion is indicated, the Blook Bank will supply UCB or RCB according to the patient's group. The primary endpoint is the incidence of any ROP. Secondary endpoints are assessessment of treatment safety, results of biomarkers related to ROP and its chronology, and urine oxidative stress markers. In addition, the cellular composition of umbilical cord blood and its relationship with prematurity-related pathologies will be analyzed. All patients will be followed-up to 24 months of corrected age to evaluate their neurodevelopment.DiscussionROP is a major cause of irreversible blindness in preterm newborns. Transfusions with adult donor blood can lead to complications, including ROP. UCB transfusions offer advantages by maintaining physiological HbF levels and potentially optimizing postnatal development. Moreover, autologous UCB transfusion could reduce risks associated with heterologous blood products, although volume collection remains challenging. UCB contains growth factors and progenitor cells that may impact ROP.

Project description:BackgroundC-peptide offers potential as a marker to indicate childhood metabolic outcomes. Measuring C-peptide concentration might have better future utility in the risk stratification of neonates born to overweight or diabetic mothers. Prior research has tried to bring this matter into the light; however, the clinical significance of these associations is still far from reach. Here we sought to investigate the associations between fetomaternal metabolic variables and umbilical cord blood C-peptide concentration.MethodsFor the present study, 858 pregnant women were randomly selected from among a sub-group of 35,430 Iranian pregnant women who participated in a randomized community non-inferiority trial of gestational diabetes mellitus (GDM) screening. Their umbilical cord (UC) blood C-peptide concentrations were measured, and the pregnancy variables of macrosomia/large for gestational age (LGA) and primary cesarean section (CS) delivery were assessed. The variation of C-peptide concentrations among GDM and macrosomia status was plotted. Due to the skewed distribution of C-peptide concentration in the sample, median regression analysis was used to identify potential factors related to UC C-peptide concentration.ResultsIn the univariate model, positive GDM status was associated with a 0.3 (95% CI: 0.06 - 0.54, p = 0.01) increase in the median coefficient of UC blood C-peptide concentration. Moreover, one unit (kg) increase in the birth weight was associated with a 0.25 (95% CI: 0.03 - 0.47, p = 0.03) increase in the median coefficient of UC blood C-peptide concentration. In the multivariate model, after adjusting for maternal age, maternal BMI, and macrosomia status, the positive status of GDM and macrosomia were significantly associated with an increase in the median coefficient of UC blood C-peptide concentration (Coef.= 0.27, 95% CI: 0.13 - 0.42, p < 0.001; and Coef.= 0.34, 95% CI: 0.06 - 0.63, p = 0.02, respectively).ConclusionUC blood concentration of C-peptide is significantly associated with the incidence of maternal GDM and neonatal macrosomia. Using stratification for maternal BMI and gestational weight gain (GWG) and investigating molecular markers like Leptin and IGF-1 in the future might lay the ground to better understand the link between metabolic disturbances of pregnancy and UC blood C-peptide concentration.

Project description:BackgroundUmbilical Cord blood (UCB), which contains a substantive number of stem cells, could be widely used in transplants to treat a variety of oncologic, genetic, hematologic, and immunodeficiency disorders. However, only a small portion of mothers preserve or donate their UCB in China. The limited availability of UCB has hampered stem cell research and therapy nowadays. To date, no systemic investigations regarding factors that influence a mother's willingness to preserve UCB have been performed in China. In the current study, we are trying to determine those factors which will provide useful information for national health policy development and will raise awareness of the importance of UCB preservation.MethodsDuring 2011 to 2013, 5120 mothers with the average age of 26.1±8.4 years were included in this study. Those mothers participated in a standardized survey. The information gathered consisted of delivery time, occupation, level of education, knowledge of preservation of UCB, willingness to store UCB, and related concerns. The results have been analyzed with SPSS 16.0.ResultsThe results showed that first-time mothers showed a greater willingness to preserve their UCB (73.3%) compared to those having their second (48.9%) or third child (40.3%). Mothers who were employed at Government Agencies and Organizations were more willing to preserve their UCB (87.3%) than those employed at factories (62.0%), and those who were unemployed (27.3%). Mothers holding master's or college degrees were more willing to preserve their UCB (72.5% and 71.1%, respectively) than mothers with high school diplomas (48.7%) or those who only went to preliminary school or middle school (40.7%). The two strongest factors that influenced an unwillingness to preserve UCB were the high cost and concerns regarding the safety of the preservation.ConclusionsThe results showed that mothers with higher education or those having better occupations are more likely to preserve their UCB in China. These mothers have related knowledge and understand the importance of the preservation and they could more readily afford the relatively high cost. The government, clinicians and UCB banks should combine efforts to take measures, such as increasing public knowledge of the importance of UCB preservation and decreasing the high cost for its storage will most likely increase the frequency of UCB preservation which will further benefit stem cell research and therapy.

Dataset Information

Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide.

Objective

Research design and methods

Results

Conclusions

Publications

Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets