Background
Autoimmune cytopenias (AICs) are rare, but serious complications of allogeneic hematopoietic cell transplantation (allo‐HSCT).
Procedure
We performed a case‐control study using 20 ...pediatric AIC cases and 40 controls, matched by stem cell source and primary indication comparing clinical and transplant characteristics, treatment, outcomes, and late effects.
Results
Cases were more likely to be human leukocyte antigen mismatched (P = 0.04). There was no difference in conditioning regimen, serotherapy use, graft‐versus‐host disease (GVHD) prophylaxis, incidence of acute or chronic GVHD, ABO compatibility, infections, and donor engraftment. The median time to AIC onset was 219 days (range, 97‐1205 days) and AIC resolution was 365 days (range, 10 days to 2737.5 days). First‐line therapies for AIC patients most commonly included corticosteroids (75%) and rituximab (55%). Only 25% of patients responded to first‐line treatment. At a median of 611.5 days from last rituximab dose, 82.5% patients were still receiving intravenous immune globulin for hypogammaglobulinemia compared with 2.5% of controls (P < 0.0001). Iron overload was higher in AIC patients (P = 0.0004), as was avascular necrosis (P = 0.04). There was no difference in overall survival at one year after HSCT (85% vs 82.5%). Two patients with refractory autoimmune hemolytic anemia responded to daratumumab and had resolution of B‐cell aplasia.
Conclusions
In this study, we find poor initial responses to AIC‐directed therapies and significant late effects.
Background
Among pediatric hematopoietic stem cell transplant (HSCT) recipients, abnormal glycemic control is shown to be associated with increased risk of transplant‐related mortality, death from ...any cause, risk of infection, increased hospitalized, and intensive care days. Independent effects of higher glycemic variability, a component of glycemic control, have not been described. This study aimed to characterize risk factors for, and consequences of, higher glycemic variability in HSCT patients.
Procedure
Medical records for a cohort of 344 patients, age 0‐30 years, who underwent first HSCT from 2007 to 2016 at Children's Hospital Colorado were retrospectively reviewed. Glucose coefficients of variation (CV) were analyzed for HSCT days −14 to 0 and 0‐30, and patients were assessed for potential risk factors and outcomes.
Results
Roughly one‐third of patients had pre‐HSCT and day 0‐30 glucose CV above the reported healthy adult range. Independent of HSCT type, doubling of pre‐HSCT glucose CV was associated with a 4.91‐fold (95% confidence interval CI, 1.40‐17.24) increased hazard of infection, as well as increased risk for intensive care hospitalization for allogenic HSCT patients. Multivariable analysis demonstrated that allogeneic HSCT patients had a 1.40‐ and 1.38‐fold (95% CI, 0.98‐1.99 and 1.00‐1.91) increased hazard of death for every doubling of pre‐HSCT and day 0‐30 glucose CV, respectively.
Conclusions
Just as with higher mean glucose, higher glycemic variability in the pediatric HSCT population is independently associated with significantly increased morbidity. Additional research is required to evaluate the utility of glucose control to mitigate these relationships and improve HSCT outcomes.
Bronchiolitis obliterans (BO) is a severe complication after allogeneic hematopoietic stem cell transplantation (1, 2).Lowattenuation scores quantify parenchymal abnormality and may be useful for ...monitoring BO as well as other diseases that feature air trapping (e.g., after lung transplantation, infectious pneumonitis of infancy, and Stevens-Johnson syndrome).
Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be ...reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and HLA-matched HPC transplantation.
We analyzed late cardiovascular outcomes of 661 patients who survived at least 2 years from hematopoietic cell transplantation for childhood hematologic malignancy between 1995 and 2008. Center for ...International Blood and Marrow Transplant Research data was supplemented with surveys focused on cardiotoxicity and potential risk factors. The median duration of follow-up was 97 months (range 24-230). 4.2% of survivors experienced at least one of the primary outcomes including coronary artery disease (0.2%), cerebrovascular accident (0.6%), cardiomyopathy (3%), and cardiac-related death (0.5%). Patients who received anthracycline chemotherapy (HR 4.67, p = 0.036) or cranial or chest radiation (HR 5.58, p < 0.0001; HR 2.18, p = 0.0087) were at increased risk for developing one of the primary outcomes. Dyslipidemia was diagnosed in 18% of survivors. Pre-transplant anthracycline (HR 1.74, p < 0.0001) and chest radiation (HR 1.34, p = 0.0371) were risk factors for dyslipidemia. Overweight/obese body mass status was present in 63% of patients at baseline, 65% at 2 years, and 52% at most recent evaluation. Diabetes was diagnosed in 7% of subjects. In conclusion, severe cardiovascular complications were infrequently reported. The incidence of risk factors including obesity and dyslipidemia were significant and will likely increase the risk of cardiovascular disease over time in transplant survivors.
Purpose The incidence of cytomegalovirus (CMV) retinitis in the pediatric allogeneic hematopoietic stem cell transplant (HSCT) population is unknown. We report a cluster of 5 pediatric patients with ...CMV retinitis diagnosed in a 12-month period, and compare this to the rate of CMV viremia and retinitis in the four years prior. Presented is the ophthalmic screening protocol developed in response to this experience. Design Retrospective cross-sectional study. Methods A retrospective chart review was performed on patients at Children’s Hospital of Colorado (CHCO) who received allogeneic HSCT between January 2010 and December 2014. Fisher Exact test was used to compare the proportion of CMV viremia and CMV retinitis in patients transplanted between January 2010 and December 2013 with those transplanted in 2014. Results 101 patients underwent allogeneic HSCT from January 2010 to December 2013; 32 (32%) tested positive for CMV viremia. No cases of CMV retinitis were identified. From January 2014 to December 2014, 28 patients underwent allogeneic HSCT; 13 patients (46%) had CMV viremia, not a statistically significant increase (p=0.18). There were five cases of CMV retinitis diagnosed in those transplanted in 2014, a statistically significant increase compared to those transplanted in 2010-2013 (p=0.0004). A multidisciplinary team was formed to review the literature and an ophthalmic screening protocol was developed. Conclusion Our recent cluster of CMV retinitis in pediatric allogeneic HSCT patients may suggest a rise in incidence of CMV retinitis. We propose an ophthalmic screening protocol to diagnose retinitis in pediatric HSCT patients in the early, often asymptomatic stage.
PDF
