The Areal Project: How Virtual Reality Application Could Enhance Patient's Quality Time during Transfusion Therapy in Adult Patients with Thalassemia and Sickle Cell Disease

Growth retardation is common in children with SCA, in spite of adequate dietary intakes. Previous studies have demonstrated elevated REE in children with SCA. An increased metabolic rate may be secondary to rapid hemolysis of sickled cells and the compensatory erythropoietic activity. The purpose of the current investigation was to determine whether the elevated REE in children with SCA can be normalized by RBC transfusion therapy. Five children aged 12 to 16 years (4 males, 1 female) were studied before and one week after a scheduled RBC transfusion. Measurements included anthropometics (height, weight), REE by indirect calorimetry, hemoglobin, reticulocyte count, and percent HgS. Growth failure was seen in 3 of 5 subjects who were below the 25% ile by NCHS standards for height. Pre-transfusion REE was significantly elevated above predicted REE (28%, p≤0.04, Wilcoxon matched-pairs signed-ranks test) consistent with reports in the literature. Surprisingly, REE increased further after RBC transfusion to 37% on average above predicted REE (pre versus post REE, p≤0.08) despite a significant decrease in erythropoietic activity (reticulocyte count: Δ = -9.3%, p≤0.04). Four of 5 subjects showed an increase in REE after RBC transfusion. The subject who had no change in REE demonstrated an increase one week after receiving a second RBC transfusion. Anemia improved after RBC transfusion with an increase in hemoglobin (Δ=1.5 g/dl, p≤0.04), and decrease in Hgb S (Δ=-31.4%, p≤0.04). These results suggest that REE is not correlated with erythropoietic activity in children with SCA and may, in fact, increase when RBC transfusion decreases erythropoietic activity. A possible explanation for this increase includes the hypothesis that a relatively hypoxic catabolic hypermetabolism exists prior to RBC transfusion that transitions in children with SCA to a more hypermetabolic anabolic state after RBC transfusion. If proven, this might explain the catch-up growth observed in children with SCA who receive chronic RBC transfusion therapy.

Middle Cerebral Artery Velocities Are Inversely Related to Hemoglobin Levels and Acutely Drop in Response to RBC Transfusion: Implications for Stroke Screening in SCD

Toxic Unbound Iron and Membrane Injury in b-Thalassemia and Sickle Cell Disease: Elevated Non-Transferrin Bound Iron (NTBI) and Malondialdehyde (MDA).

A 21-year-old male with sickle cell disease (SCD) presented with severe pallor. He had received a total of 100 red blood cell (RBC) units in his lifetime, had a mean serum ferritin level of 3133 ng/ml, and liver iron concentration (LIC) of 12 mg Fe/g dry weight (dw). He was started on subcutaneous deferoxamine (DFO) infusions at a dose of 56 mg/kg/d, five days a week (equivalent to 40 mg/kg/d, seven days a week) and continued to receive 8–10 RBC units/year as treatment for pain. During the first six months of chelation therapy, his serum ferritin levels fell by around 50% of the pretreatment value, but then started to increase back up to the baseline values. The patient was noncompliant with DFO therapy. He experienced pain at the site of injection, could not sleep and was concerned about carrying a pump and not being accepted by his peers. He dropped out of college and abstained from all social activities. He was referred to a psychologist; however, this failed to improve compliance and he opted to stop DFO therapy altogether.

Quality of Care in United States Children with Sickle Cell Anemia

The Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Sickle Cell Disease and Chronic Kidney Disease: A Randomized, Multicenter, Open-Label, Phase II Study (STEADFAST)

Health Care Utilization in Transplanted Versus Non-Transplanted Sickle Cell Disease Patients

Global Longitudinal Myocardial Strain Correlates with Degree of Anemia in Sickle Cell Disease but Not Transfusion-Dependent Thalassemia

Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy

Effects of Combination Hydroxyurea and Chronic Red Blood Cell Transfusion Therapy on the Immune Parameters of Patients with Sickle Cell Disease

Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy

Normalization of cerebral hemodynamics after hematopoietic stem cell transplant in children with sickle cell disease

Clinical effects of different types of red cell concentrates in patients with thalassemia and sickle cell disease

Determining how the health-related quality of life (HRQOL) is impacted by living with Sickle Cell Disease (SCD) can inform psychosocial interventions. The purpose of the present study is to determine if demographic and treatment variables predict membership into empirically derived subgroups of HRQOL among youth and young adults with SCD. Three hundred and seven youth and young adults with SCD (mean 17.63 years ± 3.74 years, 50.5% female) completed the Pediatric Quality of Life InventoryTM Sickle Cell Disease Module. Latent profile analysis examined subgroups/classes of HRQOL and relationships with demographic and treatment variables. Three distinct classes emerged: High HRQOL (34% of the sample), Moderate HRQOL (44% of the sample), and Low HRQOL (22% of the sample). Being female was associated with increased odds of being in the moderate or low groups. Living with more severe SCD (genotypes HbSS and HbSβ0 thalassemia) was associated with increased odds of being in the Low HRQOL group. Treatment with chronic red blood cell transfusion therapy was associated with increased odds of being in the High HRQOL group. Older age predicted a small increase in the odds of being in the Low versus High HRQOL group. The present study adds to the literature on HRQOL in SCD by exploring person-centered, empirically derived groups of HRQOL. Identification of demographic and treatment factors that predict membership into those groups within a large sample assists in tailoring needed psychosocial interventions for youth with SCD.

Hematopoietic Cell Transplantation in Thalassemia and Sickle Cell Disease: Report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000-2017