Patient reported outcomes (PROs) provide multidimensional perspectives on the impact of pain1, 2, 3, 4, 5 and have been assessed using the Patient Reported Outcomes Measurement Information System (PROMIS) in children with sickle cell disease (SCD) during outpatient visits,3,4 acute care utilization for pain,4,6 and in clinical trials to measure the effect of interventions.7, 8, 9 Here, we report the feasibility of collection of PROs of pain interference and fatigue at enrollment (baseline) from participants 3 to 21 years of age enrolled in a phase 2 randomized double-blind placebo-controlled trial of intravenous arginine therapy for acute SCD pain (NCT02536170),10 and examine the impact of an acute pain episode on these PROs.

Acute, intermittent vaso-occlusive pain is the hallmark of sickle cell disease (SCD) and is associated with substantial morbidity and impaired quality of life (QOL). The subgroup of adults with SCD who transition from recurrent, acute pain to chronic, persistent pain have even greater QOL impairment and higher rates of healthcare utilization. Self-management is central to SCD management; however, its role in chronic pain management is not established. This qualitative study was conducted to answer the following research questions: (1) What is the chronic pain experience of adults with SCD? (2) What self-management strategies do adults with SCD use for chronic pain? and (3) Do adults with SCD have any needs in the self-management of chronic pain? Eighteen Black adults with SCD completed a demographics questionnaire and an interview. The majority of the participants were 21–30 years of age (mean 33.5, SD 7.6), female (61.1%), employed at least part-time (61.1%), single/never married (72.2%), and had a SCD type of sickle cell anemia (55.5%). Interview analysis revealed three major themes: (1) the chronic pain experience; (2) strategies for managing chronic pain; and (3) challenges and needs in managing chronic pain. Study findings can be used to support chronic pain management among adults with SCD. Further research is needed to devise and implement effective strategies for the prevention and management of chronic SCD pain.

Sickle cell disease (SCD) is the most common hemoglobinopathy in the United States and vaso-occlusive pain episodes (VOE) are the leading cause of hospitalizations and emergency department (ED) visits1. There are limited therapies for acute management of VOE that directly target the underlying etiology for sickle-related pain therefore treatment is largely symptomatic. Moderate-to-severe pain is typically treated with parenteral hydration, opioids, and hospitalization to achieve adequate pain control.

Sickle cell disease (SCD) is an inherited hemoglobinopathy associated with severe morbidity, impaired quality of life, and premature mortality. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available for patients with SCD and has a >90% event-free survival when a matched related donor is used. However, availability of human leukocyte antigen (HLA)effidentical sibling donors for the SCD population is limited. The use of HLA-matched unrelated donors or related haploidentical donors has the potential to expand the donor pool. Methods: We reviewed the current literature on the indications for SCD transplantation, donor options, and the emerging use of gene therapy as a treatment option. Google Scholar and PubMed were searched using the terms SCD, bone marrow transplantation, donor sources, gene therapy, HSCT, and HLAmatching. Additional articleswere identified fromthe bibliographies of retrieved articles. All articles were reviewed for pertinent information related to SCD and transplantation. Results: HSCT has the potential to establish donor-derived normal erythropoiesis with stable long-term engraftment, amelioration of symptoms, and stabilization of organ damage. The majority of HSCT has been performed in children fromHLA-identical sibling donors and has resulted in excellent rates of survival. The use of alternate donors such as HLA-matched unrelated donors and haploidentical donors has the potential to expand the applicability of HSCT for SCD. Early results in gene therapy for SCD are encouraging.