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Annals of Internal Medicine Feb 2024Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage...
BACKGROUND
Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community.
OBJECTIVE
To evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs).
DESIGN
Comparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases.
DATA SOURCES
Centers for Medicare & Medicaid Services claims data, 2008 to 2016; published literature.
TARGET POPULATION
Persons eligible for gene therapy.
TIME HORIZON
Lifetime.
PERSPECTIVE
U.S. health care sector and societal.
INTERVENTION
Gene therapy versus common care.
OUTCOME MEASURES
Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves.
RESULTS OF BASE-CASE ANALYSIS
At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values.
RESULTS OF SENSITIVITY ANALYSIS
Results were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival.
LIMITATION
The short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study.
CONCLUSION
Gene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis.
PRIMARY FUNDING SOURCE
National Heart, Lung, and Blood Institute.
Topics: Aged; Male; Humans; United States; Cost-Effectiveness Analysis; Quality of Life; Cost-Benefit Analysis; Medicare; Anemia, Sickle Cell; Quality-Adjusted Life Years
PubMed: 38252942
DOI: 10.7326/M23-1520 -
International Journal of Impotence... Feb 2024Conservative and medical treatments are considered the first step in ischemic priapism (IP) management, although there is no clear evidence regarding their efficacy. We... (Review)
Review
Conservative and medical treatments are considered the first step in ischemic priapism (IP) management, although there is no clear evidence regarding their efficacy. We conducted a systematic review on behalf of the EAU Guidelines panel on Sexual and Reproductive health to analyse the available evidence on the efficacy and safety of conservative and medical treatment for non-sickle cell disease-related IP. Databases searched for relevant literature investigating efficacy and safety of conservative measures and medical treatment for IP included Medline, EMBASE, Cochrane Libraries and clinicaltrial.gov published up to September 2021. Overall, 41 retrospective, 3 prospective single-arm studies and 3 randomized controlled trials met the inclusion criteria. Intracavernous injection with sympathomimetic (ICIs) agents were the most frequently utilized treatment with efficacy ranging from 0 to 100% of cases. The combination of ICIs with corporeal aspiration with or without irrigation with saline was successful in 70 to 100% of cases. Oral treatment with β2 receptor agonist (e.g., terbutaline) showed mild to moderate efficacy. Conservative methods including ice pack, exercise, cold enema and ejaculation depicted lower effectiveness in resolving priapism (1-55%). Longer time interval from the onset to the resolution of IP was associated with higher rate of erectile dysfunction at follow-up (30-70%), especially after 24 h.
Topics: Male; Humans; Priapism; Retrospective Studies; Prospective Studies; Reproductive Health; Erectile Dysfunction
PubMed: 35995858
DOI: 10.1038/s41443-022-00592-2 -
BJUI Compass Jan 2024To evaluate clinical characteristics associated with survival in patients with metastases to the penis.
OBJECTIVES
To evaluate clinical characteristics associated with survival in patients with metastases to the penis.
METHODS
After approval by the IRB, records of collaborating centres in Leuven, London, Rostock, Amsterdam and Tampa were screened for men presenting with metastatic disease to penis. Multivariate logistic regression analyses were used to identify covariables associated with survival. We analysed clinical data on 34 patients.
RESULTS
Primary sites were most frequently prostate ( = 14, 41%) and bladder ( = 9, 26%). Twenty-eight of 34 (82%) presented with metachronous penile metastases, and 11 (32%) patients had penile metastases as the sole metastatic site. Penile metastatic locations were most frequently in the corpora ( = 18; 53%). Seven (21%) patients with penile metastases had priapism on presentation. Systemic therapy was frequent and variable (chemotherapy = 12; immunotherapy = 5; hormones = 3). Local management included either surgery ( = 10) or RT ( = 8). Twelve- and 24-month overall survival rate were 67% and 35%, respectively. No clinical parameter including primary histology, synchronous or metachronous metastases or priapism showed statistical survival benefit or detriment.
CONCLUSION
Metastasis to penis arises most frequently from pelvic primaries. Priapism does not appear to correlate with survival in this large, well-defined series.
PubMed: 38179026
DOI: 10.1002/bco2.282 -
Urology Research & Practice Nov 2023Malignant priapism (MP) is defined as a condition of persistent erection of the penis without sexual stimulation due to the neoplastic process of the cavernous sinus and...
Malignant priapism (MP) is defined as a condition of persistent erection of the penis without sexual stimulation due to the neoplastic process of the cavernous sinus and the efferent veins. The effectiveness of established therapeutic recommendations in priapism was ineffective in MP. Modalities of therapy for MP varied from medication treatment, nonsurgical treatment, surgical treatment, and radiotherapy. Despite aggressive surgical management combined with radiation therapy, chemotherapy, or targeted therapy, the survival rate remains low. Therefore, the treatment is usually palliative, focusing on the patient's quality of life improvement and symptom relief.
PubMed: 37971391
DOI: 10.5152/tud.2023.23088 -
International Journal of Impotence... Feb 2024
Review
ManAgement of pRiapiSm and its impact on outcomes: an international register (MARS study) - the first international, multicenter, observational study regarding priapism in perspective.
PubMed: 38418865
DOI: 10.1038/s41443-024-00849-y -
Advanced Emergency Nursing JournalThis article presents a case study focusing on priapism in a patient with sickle cell disease, with repeated emergency department (ED) visits and hospitalizations. The...
This article presents a case study focusing on priapism in a patient with sickle cell disease, with repeated emergency department (ED) visits and hospitalizations. The patient was successfully identified and treated by the ED nurse practitioner (NP) with aspiration of the corpus cavernosum. Priapism is a persistent penile erection that continues for an extended time. There is some argument about what that length of time is, but generally, the consensus is more than 4 hr beyond sexual stimulation or unrelated to sexual stimulation or sexual interest (Bivalacqua et al., 2022). Priapism is a fairly common but underrecognized complication of sickle cell disease. It represents a urological emergency in which timely diagnosis and appropriate treatment are vital to preserving penile tissue and sexual function. The diagnosis is made clinically with a comprehensive history, physical examination, and appropriate laboratory test values. Initial management can be conservative with hydration and analgesics or, if necessary, more invasive with needle aspiration to promote detumescence. Permanent tissue damage or erectile dysfunction can result if priapism is unrecognized, untreated, or not treated immediately. The NP plays an integral role in treating and preventing permanent damage. Patient education should focus on instructions for preventing priapism and managing episodes at home.
Topics: Male; Young Adult; Humans; Priapism; Anemia, Sickle Cell; Consensus; Emergency Room Visits; Hospitalization
PubMed: 38285418
DOI: 10.1097/TME.0000000000000494 -
Sexual Medicine Reviews Mar 2024Penile induration disease, commonly known as Peyronie's disease (PD), is a connective tissue disorder that affects the penis, leading to the development of fibrous...
INTRODUCTION
Penile induration disease, commonly known as Peyronie's disease (PD), is a connective tissue disorder that affects the penis, leading to the development of fibrous plaques, penile curvature, and erectile dysfunction. PD is a common male reproductive system disease with a complex etiology involving multiple genes, signaling pathways, and different phenotypes.
OBJECTIVES
The etiology and pathogenesis of PD remain poorly understood, hindering the development of effective treatment strategies. By understanding the underlying mechanisms of PD, we can pave the way for targeted therapies and improved patient outcomes.
METHODS
We reviewed the epidemiology and pathophysiology of PD. We performed database searches on Google Scholar, PubMed, Medline, and Web of Science from inception to September 2023. The literature reviewed included priapism guidelines, review articles, current trial studies, and various literature related to PD.
RESULTS
This article provides a comprehensive overview of the current research progress on the disease, focusing on its genetic factors, signaling pathways, cellular mechanisms, phenotypic manifestations, and therapeutic targets. It can help identify individuals at higher risk, aid in early detection and intervention, and provide insights into fibrosis and tissue remodeling. It can also reveal potential therapeutic targets, guide accurate diagnoses and treatment strategies, and address the impact of the disease on patients' quality of life.
CONCLUSION
By integrating insights from genomics, molecular pathways, clinical phenotypes, and therapeutic potentials, our research aims to achieve a deeper and more comprehensive understanding of PD, propelling the field toward innovative strategies that enhance the lives of those affected by PD. The complex manifestations and pathogenesis of PD necessitate the use of multiple treatment methods for personalized care.
PubMed: 38456235
DOI: 10.1093/sxmrev/qeae006 -
Orthopedic NursingPriapism is a disorder where the penis without sexual stimulation maintains a prolonged rigid erection lasting 4 or more hours. There are two classifications of... (Review)
Review
Priapism is a disorder where the penis without sexual stimulation maintains a prolonged rigid erection lasting 4 or more hours. There are two classifications of priapism, ischemic (low flow) or nonischemic high flow, and each have specific etiologies, diagnostic criteria, and management. This presented case study involved a 58-year-old male who experienced an ischemic priapism more than 24 hours after an anterior lumbar interbody fusion (ALIF). A flaccid penis was achieved after the patient received two 400 µg of phenylephrine HCL into the corpora cavernosum. Review of the literature suggests anesthetic medications given during the surgical procedure may have caused the priapism. Lessons that can be learned from this case study highlight that even though the nurse may not expect to see a priapism after an ALIF, the nurse must always be diligent and not become complacent with unexpected findings or assessments that may cause irreparable harm to the patient.
Topics: Male; Humans; Middle Aged; Priapism; Learning; Lumbosacral Region; Phenylephrine
PubMed: 38266263
DOI: 10.1097/NOR.0000000000001001 -
Undersea & Hyperbaric Medicine :... 2023The United States Navy (USN) developed and refined standardized oxygen treatment tables for diving injuries, but USN tables may not address all situations of spinal...
INTRODUCTION
The United States Navy (USN) developed and refined standardized oxygen treatment tables for diving injuries, but USN tables may not address all situations of spinal decompression sickness (DCS). We describe a detailed recompression treatment regimen that deviated from standard USN protocol for an active-duty USN diver with a severe, delayed presentation of spinal cord DCS.
CASE REPORT
A USN diver surfaced from his second of three dives on a standard Navy 'no-Decompression' Air SCUBA dive (Max depth 101 fsw utilizing a Navy Dive Computer) and developed mid-thoracic back pain, intense nausea, paresthesias of bilateral feet, and penile erection. Either not recognizing the con- stellation of symptoms as DCS and after resolution of the aforementioned symptoms, he completed the third planned dive (essentially an in-water recompression). Several hours later, he developed paresthesias and numbness of bilateral feet and legs and bowel incontinence. He presented for hyperbaric treatment twenty hours after surfacing from the final dive and was diagnosed with severe spinal DCS. Based on the severity of clinical presentation and delay to treatment, the initial and follow-on treatments were modified from standard USN protocol. MRI of the spine four days after initial presentation demonstrated a 2.2 cm lesion at the T4 vertebral level extending caudally. Follow-up examinations over two years demonstrated almost complete return of motor and sensory function; however, the patient continued to suffer fecal incontinence and demonstrated an abnormal post-void residual urinary volume. An atypical presenting symptom, a discussion of MRI findings, and clinical correlations to the syndrome of spinal DCS are discussed throughout treatment and long-term recovery of the patient.
Topics: Male; Humans; United States; Decompression Sickness; Paresthesia; Diving; Hyperbaric Oxygenation; Laminectomy
PubMed: 38055878
DOI: No ID Found -
The Journal of Sexual Medicine May 2024Intracavernosal injection therapy (ICI) is a well-established therapeutic strategy for men with erectile dysfunction. Complications are often related to patient error...
BACKGROUND
Intracavernosal injection therapy (ICI) is a well-established therapeutic strategy for men with erectile dysfunction. Complications are often related to patient error when performong ICI.
AIM
The objective of this study was to examine patient errors in an established patient training program for performing ICI and identify factors that could predict major errors.
METHODS
Patients enrolled in our ICI program are trained on technical aspects, and dose titration is begun. Patients are given explicit instructions during training, both verbally and in written form. Records were reviewed for men using ICI for ≥6 months. Multivariable analysis was used to define predictors of major errors.
OUTCOMES
Errors were listed as minor (zero-response injection, penile bruising, expired medication) and major (errors potentially leading to priapism: dose self-titration, double injecting).
RESULTS
Overall, 1368 patients met the inclusion criteria and were included in the analysis. The mean patient age was 66 ± 22 (range 29-91) years. Regarding education, 41% of patients had graduate-level education, 48% had college education, and 11% high school education. Mean follow-up was 3.2 ± 7.6 (range 0.5-12) years. The agents used were trimix (62%), bimix (35%), papaverine (2%), and prostaglandin E1 monotherapy (1%). At least 1 error occurred during self-administration in 42% of patients during their time in the program. Errors included zero response to medication due to technical error (8% of patients), penile bruising (34%), use of an expired bottle (18%), self-titration (5%), and double injecting (4% of patients); 12% of men committed ≥1 error during their time in the program. On multivariable analysis, independent predictors of the occurrence of a major error included: young age, graduate-level education, and <12 months of injection use.
CLINICAL IMPLICATIONS
To the best of our knowledge, this is the first reported study to investigate ICI errors and risk factors. The identification of factors predictive of major errors allows for more tailored and intensive training in this subset of patients.
STRENGTHS AND LIMITATIONS
Strengths of this study include a large patient population (1386 men) with a considerable follow-up time. Additionally, the rigorous training, education, and monitoring of the participants, as well as the use of formal definitions, enhances the accuracy and reliability of the results. Despite the strengths of the study, recall bias may be a limitation concern.
CONCLUSION
The majority of patients were error free, and the majority of the errors were minor in nature. Major errors occurred in <10% of patients. Younger age, graduate-level education, and less experience with ICI were independent predictors of major errors.
Topics: Humans; Male; Adult; Middle Aged; Aged; Erectile Dysfunction; Aged, 80 and over; Injections; Patient Education as Topic; Vasodilator Agents; Medication Errors; Papaverine; Penis; Medical Errors; Priapism
PubMed: 38660738
DOI: 10.1093/jsxmed/qdae031