Allogeneic hematopoietic stem cell transplantation (HSCT) is rarely performed in adult patients with sickle cell disease (SCD). We utilized the chemotherapy-free, alemtuzumab/total body irradiation 300 cGy regimen with sirolimus as post-transplantation immunosuppression in 13 high-risk SCD adult patients between November 2011 and June 2014. Patients received matched related donor (MRD) granulocyte colony-stimulating factor-mobilized peripheral blood stem cells, including 2 cases that were ABO incompatible. Quality-of-life (QoL) measurements were performed at different time points after HSCT. All 13 patients initially engrafted. A stable mixed donor/recipient chimerism was maintained in 12 patients (92%), whereas 1 patient not compliant with sirolimus experienced secondary graft failure. With a median follow-up of 22 months (range, 12 to 44 months) there was no mortality, no acute or chronic graft-versus-host disease (GVHD), and no grades 3 or 4 extramedullary toxicities. At 1 year after transplantation, patients with stable donor chimerism have normalized hemoglobin concentrations and improved cardiopulmonary and QoL parameters including bodily pain, general health, and vitality. In 4 patients, sirolimus was stopped without rejection or SCD-related complications. These results underscore the successful use of a chemotherapy-free regimen in MRD HSCT for high-risk adult SCD patients and demonstrates a high cure rate, absence of GVHD or mortality, and improvement in QoL including the applicability of this regimen in ABO mismatched cases (NCT number 01499888).

Studies have consistently evidenced the positive clinical, economic, and humanistic benefits of pharmacist-directed patient care in a variety of settings. Given the vast differences in clinical outcomes associated with evaluated clinical pharmacy services (CPS), more detail as to the nature of the CPS is needed to better understand observed differences in economic outcomes. With the growing trend of outpatient pharmacy services, these economic evaluations serve as viable decision-making tools in choosing the most effective and cost-effective pharmacy programs. We previously conducted three systematic reviews to evaluate the economic impact of CPS from 1988 to 2005. In this systematic review, our objectives were to describe and evaluate the quality of economic evaluations of CPS published between 2006 and 2010, with the goal of informing administrators and practitioners as to their cost-effectiveness. We searched the scientific literature by using the Medline, International Pharmaceutical Abstracts, Embase, and Cumulative Index to Nursing and Allied Health Literature databases to identify studies describing CPS published from 2006 to 2010. Studies meeting our inclusion criteria (original research articles that evaluated CPS and described economic and clinical outcomes) were reviewed by two investigators. Methodology used, economic evaluation type, CPS setting and type, and clinical and economic outcome results were extracted. Results were informally compared with previous systematic reviews. Of 3587 potential studies identified, 25 met inclusion criteria. Common CPS settings were hospital (36%), community (32%), and clinic or hospital-based ambulatory practices (28%). CPS types were disease state management (48%), general pharmacotherapeutic monitoring (24%), target drug programs (8%), and patient education (4%). Two studies (8%) listed CPS as medication therapy management. Costs were evaluated in 24 studies (96%) and sufficiently described in 13 (52%). Clinical or humanistic outcomes were evaluated in 20 studies (80%) and were sufficiently described in 18 (72%). Control groups were included in 16 (70%) of 23 studies not involving modeling. Study assumptions and limitations were stated and justified in eight studies (32%). Conclusions and recommendations were considered justified and based on results in 24 studies (96%). Eighteen studies (72%) involved full economic evaluation. The mean ± SD study quality score for full economic evaluations (18 studies) was 60.4 ± 22.3 of a possible 100 points. Benefit-cost ratios from three studies ranged from 1.05:1 to 25.95:1, and incremental cost-effectiveness ratios of five studies were calculated and reported. Fewer studies documented the economic impact of CPS from 2006-2010 than from 2001-2005, although a higher proportion involved controlled designs and were full economic evaluations. Evaluations of ambulatory practices were increasingly common. CPS were generally considered cost-effective or provided a good benefit-cost ratio.

Background There are currently no standard diagnostic criteria for characterizing advanced Parkinson’s disease (APD) in clinical practice, a critical component in determining ongoing clinical care and therapeutic strategies, including transitioning to device-aided treatment. The goal of this analysis was to determine the proportion of APD vs. non-advanced PD (non-APD) patients attending specialist PD clinics and to demonstrate the clinical burden of APD. Methods OBSERVE-PD, a cross-sectional, international, observational study, was conducted with 2615 PD patients at 128 movement disorder centers in 18 countries. Motor and non-motor symptoms, activities of daily living, and quality-of-life end points were assessed. The correlation between physician’s global assessment of advanced PD and the advanced PD criteria from a consensus of an international group of experts (Delphi criteria for APD) were evaluated. Results According to physician’s judgment, 51% of patients were considered to have APD. There was a moderate correlation between physician’s judgment and Delphi criteria for APD (K = 0.430; 95% CI 0.406–0.473). Activities of daily living, motor symptom severity, dyskinesia duration/disability, “Off” time duration, non-motor symptoms, and quality-of-life scores were worse among APD vs. non-APD patients ( p < 0.0001 for all). APD patients (assessed by physicians) had higher disease burden by motor and non-motor symptoms compared with non-APD patients and a negative impact on activities of daily living and quality of life. Conclusions These findings aid in identifying standard APD classification parameters for use in practicing physicians. Improvements in identification of APD patients may be particularly relevant for optimizing treatment strategies including transitioning to device-aided treatment. Electronic supplementary material The online version of this article (10.1186/s12883-019-1276-8) contains supplementary material, which is available to authorized users.

BackgroundLevodopa‐carbidopa intestinal gel (LCIG; carbidopa‐levodopa enteral suspension in the United States), delivered via percutaneous gastrojejunostomy (PEG‐J) and titrated in the inpatient setting, is an established treatment option for advanced Parkinson's disease (PD) patients with motor fluctuations. However, long‐term prospective data on the efficacy of LCIG on non‐motor symptoms and the safety of outpatient titration are limited.MethodsIn this 60‐week, open‐label phase 3b study, LCIG titration was initiated in an outpatient setting following PEG‐J placement in PD patients. The efficacy of LCIG on motor and non‐motor symptoms, quality of life, and safety was assessed.ResultsThirty‐nine patients were enrolled in the study and 28 patients completed the treatment. A majority of patients (54%) completed outpatient titration within the first week of LCIG infusion. LCIG led to significant reductions from baseline in Non‐Motor Symptom Scale (NMSS) total score (least squares mean ± SE = −17.6 ± 3.6, P < 0.001) and 6 of the NMSS domain scores (sleep/fatigue, attention/memory, gastrointestinal tract, urinary, sexual function, miscellaneous) at week 12. These reductions were maintained at week 60 with the exception of the urinary domain. “Off” time (−4.9 ± 0.5 hours/day, P < 0.001) and “On” time without troublesome dyskinesia (−4.3 ± 0.6 hours/day, P < 0.001) were improved at week 60. Adverse events (AEs) were reported in 37 (95%) patients.Conclusions LCIG treatment led to reductions in non‐motor symptom burden and motor fluctuations in advanced PD patients. The safety profile was consistent with previous studies that used inpatient titration and outpatient titration did not appear to pose additional risk.

LCIG is a cost-effective treatment option compared with SoC in patients with aPD.