Functional Outcomes of Children with Sickle Cell Disease Treated for Vaso-Occlusive Painful Events.
Authors: Brandow Amanda M. , Brousseau David C. , Panepinto Julie A. 
Published: October 2019

Vaso-occlusive painful events lead to frequent Emergency Department (ED) visits and hospitalizations for children with sickle cell disease. After treatment to alleviate the acute pain, these children are discharged to home, however, the degree of functional impairment once children go home is not known. The objective of this study was to describe functional outcomes of children post discharge from a vaso-occlusive painful event. We conducted a prospective cohort study over a one year period of children with sickle cell disease ages 2–18 years presenting to the ED with an acute vaso-occlusive event defined as pain, acute chest syndrome or priapism. Functional outcomes were defined for this study as days of persistent pain (child and/or caretaker report), missed days of school for the child and missed days of work for the caretaker post discharge from the ED or hospital. These were assessed at both 3 and 7 days post discharge via follow-up telephone calls. Descriptive statistics were applied and linear regression was used to examine the effect of age, length of stay and history of prior vaso-occlusive events on functional outcomes post discharge. A total of 58 patients were enrolled. The mean age (SD) was 10.8 years (4.72), 52.5% were female. Genotype of sickle cell disease was 65.6% HgbSS, 24.6% HgbSC, 6.6% HgbSB+thal, and 3.3% other. There were 23% of patients discharged from the ED. Mean length of stay (SD) was 2.16 days (1.83). There were 24.1% of patients who had 3 or more hospitalizations for vaso-occlusive painful events in the year prior to study entry. Functional outcomes post discharge for all patients revealed that both child and caretaker on average missed more than one day of work and children had more than two days of persistent pain (Table 1). Subgroup analysis showed that patients with frequent prior vaso-occlusive events had significantly worse outcomes in regards to days of persistent pain and missed days of school post discharge (Table 1). Of those children attending school, 28.6% missed 3 or more days of school post discharge. Of those caretakers working, 9.1% missed 3 or more days of work post discharge. Persistent pain was reported by 15.5% of children at 5 or more days post discharge. Linear regression showed that a history of frequent vaso-occlusive painful events predicted worse functional outcomes (p=0.0067). Vaso-occlusive painful events have an enormous functional impact post discharge on both the child and family. Children with a history of frequent vaso-occlusive painful events have worse functional outcomes, and are therefore a high risk patient group. Since vaso-occlusive painful events are the most common complications of sickle cell disease, interventions to improve functional outcomes at home in the post discharge period are imperative for all patients with sickle cell disease and in particular this high risk group.

Table 1. Functional Outcomes Outcome All Patients (n=58)* Patients with < 3 Prior Vaso-occlusive Events (n=44)*† Patients with ≥ 3 Prior Vaso-occlusive Events (n=14)*† Comparison of Means between <3 and ≥ 3 Prior Vaso-occlusive Events§ *Mean ± Standard Deviation; †In year prior to study entry; §Student’s t-test Missed Days of School/Work     Child (school) 1.83 (±1.32) 1.61 (±1.07) 3.6 (±1.34) p=0.001     Caretaker (work) 1.15 (±1.20) 1.28 (±1.28) 1.0 (±0.89) p=0.617 Days of Persistent Pain 2.53 (±2.01) 2.0 (±1.41) 3.86 (±2.63) p=0.023