Incorporating integrative medicine and patient preferences into a pilot interdisciplinary sickle cell wellness clinic.

Objective: Sickle Cell Disease (SCD) is an inherited blood disorder that includes acute pain episodes and chronic pain that can dramatically impact quality of life and goal-achievement. Our staff had limited success in connecting families with the Pain, Palliative Care and Integrative Medicine Clinic (PPCIM) to receive specialized skills for pain management. We created a partnership between Hematology and PPCIM to provide SCD patients/families with needed resources.

End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.

To address the global burden of sickle cell disease and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to patient-reported outcome, pain (non-patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.

Hydroxyurea use in young infants with sickle cell disease.

Background: Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age.

Procedures: We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016.