Baseline Characteristics of Patients with Sickle Cell Disease in An Ongoing 5-Year, Prospective, Noninterventional Registry Trial

Abstract

Abstract 1060▪▪This icon denotes a clinically relevant abstract Introduction:The baseline characteristics and therapeutic management of sickle cell disease (SCD) can be quite variable depending on disease severity. This report describes baseline assessments and therapies of 420 patients in an ongoing registry designed to study current treatment patterns, natural history, and outcomes in patients with SCD. Methods:This registry trial involving 57 US hematology centers had a sequential recruitment target of 600 patients with SCD subdivided into 3 age groups (2-<12 yr, 12-<18 yr, ≥18 yr). Patients ’2 years of age with HbSS, HbS/β-thalassemia, or HbSC were eligible. Follow-up visits were every 6 months for a maximum of 5 years. Assessments included demographics, SCD history, Karnofsky Performance Status, medical history, types of crises, frequency of crises and hospitalization, treatment history, transfusion practices and use of iron chelation. Differences between pediatric (<18 yrs) and adult (≥18 yrs) patients were examined. (ClinicalTrials.gov identifier, NCT01220115). Results:Enrollment: 420 patients (254 <18 yrs, 166 ≥18 yrs) completed the initial baseline visit. Baseline characteristics and medical history are shown in the Table and Figure.Table:Baseline characteristics of patients enrolled in registry trial<18yrs (N=254) n (%)*≥18yrs (N=166) n (%)*Age, yrs – mean±SD8.9 ± 4.535.5 ± 12.4Sex, M:F – %58:43:0048:52:00Karnofsky Performance Status98 (38.6)23 (13.9)100%69 (27.2)50 (30.1)90%23 (9.1)56 (33.7)≤80%Type of SCD193 (76.0)117 (70.5)HbSS27 (10.6)16 (9.6)HbS/β-thalassemia32 (12.6)33 (19.9)HbSCTransfusions54 (21.3)29 (17.5)Regular (≥6/yr)58 (22.8)49 (29.5)Intermittent (<6/yr)134 (52.8)73 (44.0)No transfusions in last 12 monthsSerum ferritin, ng/ml – Median (range)435 (15–10054)702.5 (6–19362)Liver iron concentration: biopsy, mg/g – Median (range)7.068 (1.070–27.115)–Chelation, patients receiving in their lifetime56 (22.0)52 (31.3)Hospitalizations0.80 (0.2–4.4)0.60 (0.2–5.2)Number/yr/patient – median (range)218 (85.8)122 (73.5)Patients hospitalized in last 5 yearsConcomitant medical history27 (10.6)15 (9.0)Aplastic episode77 (30.3)23 (13.9)Asthma/reactive airway disease7 (2.8)48 (28.9)Avascular necrosis, hip(s)/shoulder(s)20 (7.9)5 (3.0)CNS disease, abnormal TCD14 (5.5)11 (6.6)CNS disease, seizure19 (7.5)9 (5.4)CNS disease, silent infarct8 (3.1)9 (5.4)CNS disease, TIA60 (23.6)12 (7.2)Dactylitis (hand food syndrome)35 (13.8)71 (42.8)Gall bladder disease0 (0.0)15 (9.0)Leg ulcer2 (0.8)13 (7.8)Lung disease, pulmonary hypertension57 (22.4)14 (8.4)Splenic sequestrationHydroxyurea use101 (39.8)64 (38.6)*n (%) unless otherwise indicated. [Display omitted] Conclusions:These data indicate differences in baseline characteristics and treatment patterns between adult and pediatric patients with SCD in this registry. Karnofsky performance status was worse, and serum ferritin levels were higher in adult versus pediatric patients. The percentage of patients having regular transfusions was higher in pediatric patients, while adult patients had higher rates of intermittent transfusions. As expected, the lifetime chelation exposure was more frequent in adult patients. Hospitalizations and use of hydroxyurea were also greater in pediatric patients. In terms of medical history related to SCDs, pediatric patients had higher frequencies of asthma, abnormal TCDs, dactylitis and splenic sequestration, while adults had more frequent evidence of avascular necrosis, gall bladder disease, leg ulcers and pulmonary hypertension. Pain was the most common crisis type in both cohorts, although this accounted for a greater relative proportion of crises in the adult cohort. The relative proportion of ACS events was lower in the adult versus pediatric patients; all other crisis types occurred in similar relative proportions in both cohorts. This ongoing registry will provide information about disease patterns, current treatment practices, and outcomes, thus contributing to a better understanding of the appropriate therapeutic management of patients with SCD. Disclosures:Heeney:Novartis: Consultancy, Research Funding. Baltz:Novartis: Research Funding. Paley:Novartis: Employment. Esposito:Novartis: Employment. Sharma:Novartis: Employment. Vichinsky:Novartis, Apotex, Ferrokin: Research Funding.