Cost-Effectiveness of Parathyroid Cryopreservation and Autotransplantation.

Importance: Delayed autotransplantation of cryopreserved parathyroid tissue (DACP) is the only surgical treatment for permanent postoperative hypoparathyroidism. Studies suggest that only a small minority of cryopreserved samples are ultimately autotransplanted with highly variable outcomes. For these reasons, many have questioned the economic utility of the process, although, to the authors' knowledge, this has never been formally studied. Objective: To report the clinical outcomes of parathyroid cryopreservation and DACP at a large academic institution and to determine the cost-effectiveness of this treatment. Design, Setting, and Participants: An institutional review board-approved, retrospective review of patients at a single institution who underwent DACP over a 17-year period was conducted with a median follow-up of 48.2 months. A forward-looking cost-utility analysis was then performed to determine the economic utility of cryopreservation/DACP vs usual care (monitoring and supplementation). Patients who had parathyroid tissue in cryopreserved storage between August 2005 to September 2022 at a single-center, academic, quaternary care center were identified. Exposure: Parathyroid cryopreservation and DACP. Main Outcomes and Measures: Graft functionality, clinical outcomes, and cost utility using a willingness-to-pay threshold of $100 000 per quality-adjusted life-year (QALY). Results: A total of 591 patients underwent cryopreservation. Of these, 10 patients (1.7%; mean [SD] age, 45.6 [17.9] years; 6 male [60%]) underwent DACP. A minority of autografts (2 [20%]) were subsequently fully functional, one-half (5 [50%]) were partially functional, and 3 (30%) were not functional. The cost-utility model estimated that at a large academic center over 10 years, the additional cost of 591 patients undergoing cryopreservation and 10 patients undergoing autotransplantation would be $618 791.64 (2022 dollars) and would add 8.75 QALYs, resulting in a cost per marginal QALY of $70 719.04, which is less than the common willingness-to-pay threshold of $100 000/QALY. Conclusions and Relevance: The reimplantation rate of cryopreserved tissue was low (<2%), but when implanted, autografts were at least partially functional 70% of the time. In the first-ever, to the authors' knowledge, formal cost analysis for this treatment, results of the current model suggest that cryopreservation and autotransplantation were cost-effective compared with the usual care for hypoparathyroidism at a large, academic institution. It is recommended that each surgical center consider whether the economic and logistical commitments necessary for cryopreservation are worthwhile for their individual needs.

Hypoparathyroidism: Consequences, economic impact, and perspectives. A case series and systematic review.

BACKGROUND: Postoperative hypoparathyroidism (PH) is the most common complication after total thyroidectomy. Incidence varies from 2% to 83%, depending on the definition. OBJECTIVE: We performed a systematic review of the literature to determine the medico-economic effects of PH and update understanding of long-term consequences, morbidity, and quality of life related to hypoparathyroidism. METHODS: We considered relevant articles published between 2000 and 2020 concerning long-term consequences of PH and quality of life. All studies concerning the medico-economic assessment of PH were included. We compared data from 2018 to results in the literature. RESULTS: A proportion of 64/403 (16.8%) patients presented with transient PH during 2018, and 7/403 (1.7%) had permanent PH. Seven patients needed supplementation with alfacalcidol at 6-month follow-up. Factors predicting the need for alfacalcidol were age <45, thyroidectomy for goiter, and lymph node dissection. Additional therapy costs related to PH were €9781.10, and additional hospital costs were €230,400. We qualitatively synthesized 41 studies. Most were retrospective studies and only a few reported costs. No series assessed direct or indirect costs of postoperative PH. CONCLUSION: To our knowledge, no previous studies reported the medico-economic impact of PH. Decreasing PH associated with fluorescence usage should be considered, particularly concerning cost-effectiveness.

Burden of illness in patients with chronic hypoparathyroidism not adequately controlled with conventional therapy: a Belgium and the Netherlands survey.

PURPOSE: To determine the burden of illness in patients with not adequately controlled chronic hypoparathyroidism receiving conventional therapy in Belgium and the Netherlands. METHODS: Data were generated from a cross-sectional, two-part online survey where endocrinologists from both countries and nephrologists from Belgium were invited by phone to participate. Part 1 included collecting data on general management of patients with hypoparathyroidism. In Part 2, physicians were requested to provide data on one or two current cases of patients with chronic hypoparathyroidism not adequately controlled on conventional therapy. Data collected included aetiology of hypoparathyroidism, clinical manifestations, comorbidities, results of laboratory and other investigations used for diagnosis and screening for complications, therapy received, and physician's perception of impaired quality of life (QoL). RESULTS: Thirty-six endocrinologists and 29 nephrologists from Belgium and 28 endocrinologists from the Netherlands participated in the survey. Data included clinical symptoms, biochemical parameters, and QoL for 97 current patients with not adequately controlled chronic hypoparathyroidism on conventional therapy. Median duration of not adequately controlled hypoparathyroidism was 2.2 years, range 0.17-20.0. Most patients had neuromuscular (85%) and/or neurological (67%) symptoms, 71% had abnormal biochemical parameters, 10% were overweight, and physicians perceived that 71% had impaired QoL. Most frequently reported comorbidities included hypertension (25%), renal comorbidity (20%), diabetes mellitus (12%), and dyslipidaemia (11%). CONCLUSION: Patients with chronic hypoparathyroidism not adequately controlled on conventional therapy experience a substantial burden of illness, mainly due to persistence of symptoms and presence of multiple comorbidities.

Clinical burden and healthcare resource utilization among patients with chronic hypoparathyroidism, overall and by adequately vs not adequately controlled disease: a multi-country chart review.

Aims: To assess the real-world clinical burden and healthcare resource utilization (HRU) among patients with chronic hypoparathyroidism, overall and by adequately controlled (AC) vs not adequately controlled (NAC) disease, informed by guideline-recommended clinical management targets, including biochemistry and symptoms. Materials and methods: In this retrospective online chart review, endocrinologists in the US, Canada, the UK, France, Germany, Italy, and Spain were randomly selected to review the medical charts of adult patients with chronic hypoparathyroidism receiving calcium and activated vitamin D. Patients' demographics, disease characteristics, symptoms, comorbidities, and hypoparathyroidism-related HRU during the 1 year before the review date were assessed. Clinical burden and HRU were compared between patients with NAC and AC hypoparathyroidism. Results: Of 614 patients with hypoparathyroidism (AC, N = 442; NAC, N = 172), the mean age was 43.6 years, and the majority were female (61.6%), Caucasian (78.8%), and had post-surgical hypoparathyroidism (74.4%). Mean duration of hypoparathyroidism was 46.0 months. Hypoparathyroidism-related symptoms and comorbidities were reported in 59.4% and 46.7% of patients, respectively; 90.7% of patients had ≥1 hypoparathyroidism-related HRU event. More patients with NAC (57.6%) vs AC (42.5%) hypoparathyroidism experienced ≥1 comorbidity including calcium/phosphate imbalances, and brain, cardiovascular, metabolic, and renal disorders (all p < 0.01). More patients with NAC vs AC hypoparathyroidism incurred ≥1 hypoparathyroidism-related hospitalization (27.9% vs 16.3%) and emergency room visits (47.7% vs 38.5%), and patients with NAC vs AC hypoparathyroidism had a higher number of outpatient visits (3.6 vs 2.6; all p < 0.05), in the 1-year observation period. Limitations and conclusions: Limitations of this online chart review include possible under-estimation of disease burden, limited sample size, and the inability to rule out selection bias. Findings indicate that patients with chronic hypoparathyroidism experience substantial symptomatic and comorbid burdens resulting in frequent HRU, suggesting an unmet need, particularly in NAC disease.

Understanding the burden of illness associated with hypoparathyroidism reported among patients in the PARADOX study.

OBJECTIVE: Hypoparathyroidism is a rare disease caused by lack of parathyroid hormone (PTH) leading to hypocalcemia, hyperphosphatemia, and a variety of symptoms. This study aimed to quantify the clinical and social burden of illness from the perspective of affected patients. METHODS: A web-based instrument was developed with input from patients, clinical experts, and the Hypoparathyroidism Association. Qualifying participants were ≥18 years old, diagnosed with hypoparathyroidism for ≥6 months, and U.S. residents. Questions focused on demographics, diagnosis perceptions, current attitudes, medical management, current symptoms, acute episodes, comorbidities, personal life, and employment. RESULTS: A total of 374 adults (mean age, 49 ± 12 years; female, 85%) with hypoparathyroidism (mean duration, 13 ± 12 years; severe condition, 30.5%) completed the survey. Patients reported visiting a mean of 6 ± 8 physicians before and after their diagnosis. The majority strongly agreed with feeling unprepared to manage the condition at diagnosis (56%), that controlling their hypoparathyroidism is harder than expected (60%), and that they were concerned about long-term complications of their current medications (75%). More than 10 symptoms were experienced by 72% of patients in the preceding 12 months, despite current management regimens. Symptoms were experienced for a mean of 13 ± 9 hours/day. Hospital stays or emergency department visits were required by 79% of patients. 45% reported significant interference with their lives, 85% reported an inability to perform household activities, and 20% experienced a disease-associated change in employment status. CONCLUSION: Patients with hypoparathyroidism have a high burden of illness and experience a broad spectrum of symptoms, with a multidimensional impact on their lives.