Review
Medicine, General & Internal
Robert Sheppard Nickel, Jacqueline Y. Maher, Michael H. Hsieh, Meghan F. Davis, Matthew M. Hsieh, Lydia H. Pecker
Summary: Curative therapy for sickle cell disease can impair future fertility and the fertility outcomes are influenced by factors such as pre-transplant ovarian reserve, semen analysis parameters, and the conditioning regimen. Patients should be offered fertility preservation before treatment and receive longitudinal post-HSCT fertility care.
JOURNAL OF CLINICAL MEDICINE
(2022)
Article
Health Care Sciences & Services
Seethal A. Jacob, Roua Daas, Anna Feliciano, Julia E. LaMotte, Aaron E. Carroll
Summary: This study aims to understand the barriers to care and perceptions of telemedicine among caregivers of pediatric SCD patients in the Midwest. The study reveals common burdens faced by caregivers, barriers and facilitators of SCD care, and caregiver perceptions of the usefulness of telemedicine for SCD care.
BMC HEALTH SERVICES RESEARCH
(2022)
Article
Oncology
Xiaoqin Yang, Francois Laliberte, Guillaume Germain, Monika Raut, Mei Sheng Duh, Shuvayu S. Sen, Dominique Lejeune, Kaushal Desai, Philippe Armand
Summary: This study found that a large portion of DLBCL patients require additional therapy after first-line treatment, highlighting the significant economic burden of DLBCL patients, especially in the first year following diagnosis.
Article
Public, Environmental & Occupational Health
Lydia H. Pecker, Elizabeth Ruvalcaba, Sophie Lanzkron, Michelle N. Eakin
Summary: This qualitative study explored adults with SCD's perspectives on telemedicine during the COVID-19 pandemic and their preferences for future telemedicine care. The findings suggest that telemedicine is most suitable for follow-up care but less effective for painful crises or urgent needs. Adults with SCD expressed a desire for telemedicine to continue and expand access to care.
JOURNAL OF RACIAL AND ETHNIC HEALTH DISPARITIES
(2023)
Editorial Material
Multidisciplinary Sciences
Benjamin Plackett
Summary: Sickle-cell disease is prevalent in the Middle East, with 2.6% of the population in some areas affected by this genetic disorder. Adlette Inati, a specialist in clinical haematology at the Lebanese American University, and president of a foundation supporting sickle-cell disease patients in Lebanon, discusses the potential benefits of genetic screening in this region.
Article
Gastroenterology & Hepatology
Grace Chen, Vasantha Pedarla, Kyle D. Null, Susan E. Cazzetta, Qasim Rana Khan, David A. Schwartz
Summary: Perianal fistula (PAF), a complication of Crohn's disease (CD), has a high prevalence in the United States and is associated with increased costs and healthcare resource utilization for patients.
INFLAMMATORY BOWEL DISEASES
(2022)
Article
Oncology
Alecia Nero, Janis Bozzo
Summary: SCD causes lifelong complications and patients often face socioeconomic disadvantages. Comprehensive SCD centers are needed to coordinate care for high-risk patients. Commercial insurance may not cover novel SCD therapies, leading to inequitable access to treatment. Understanding the economic and nonfinancial aspects of SCD care is important for evaluating comprehensive care models.
HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA
(2022)
Article
Economics
David Holdford, Nicholas Vendetti, Daniel M. Sop, Shirley Johnson, Wally R. Smith
Summary: This study aimed to quantify the indirect costs of sickle cell disease in the United States and found that pain events had a significant negative impact on employment status, productivity, and daily lives of adults with the disorder.
Article
Nursing
Andreia Insabralde de Queiroz Cardoso, Marcos Antonio Ferreira, Carolina Mariano Pompeo, Caroline Neris Ferreira Sarat, Mayk Penze Cardoso, Maria Lucia Ivo
Summary: The study analyzed complete economic studies focusing on the treatment of sickle cell anemia, specifically looking at the perspectives of using hydroxyurea and blood transfusion. No studies were found in Brazil, highlighting the need for worldwide assessment of existing technologies, reassessment of current technologies, and implementation of continuous diagnosis and treatment processes.
ACTA PAULISTA DE ENFERMAGEM
(2021)
Article
Genetics & Heredity
Alyssa Grygiel, Felicia Ikolo, Raphielle Stephen, Dawnell Bleasdille, Patricia Robbins-Furman, Beverly Nelson, Andrew K. Sobering, Sarah H. Elsea
Summary: The study in Grenada revealed that SCD patients face significant barriers to healthcare and experience negative impacts on their quality of life. Additionally, caregivers of SCD patients also reported experiencing burdens. The findings highlight the importance of further research into specific healthcare delivery barriers and the need for increased education and support for individuals affected by SCD in Grenada and the wider Caribbean community.
MOLECULAR GENETICS & GENOMIC MEDICINE
(2021)
Article
Genetics & Heredity
Alyssa Grygiel, Felicia Ikolo, Raphielle Stephen, Dawnell Bleasdille, Patricia Robbins-Furman, Beverly Nelson, Andrew K. Sobering, Sarah H. Elsea
Summary: Grenada, a small resource-limited Caribbean country, faces challenges in healthcare access and quality of life for individuals with sickle cell disease (SCD). Findings indicate that both SCD patients and caregivers encounter barriers to healthcare, while adults and children with SCD experience different issues in quality of life.
MOLECULAR GENETICS & GENOMIC MEDICINE
(2021)
Article
Oncology
Ronda Copher, Arianna Kee, Aaron Gerds
Summary: Myelofibrosis results in increased resource utilization and costs for patients and the US healthcare system. This study provides insights into treatment patterns, healthcare resource utilization, and costs associated with myelofibrosis, particularly in patients treated with ruxolitinib.
Article
Multidisciplinary Sciences
Shannon Phillips, Yumei Chen, Rita Masese, Laurence Noisette, Kasey Jordan, Sara Jacobs, Lewis L. Hsu, Cathy L. Melvin, Marsha Treadwell, Nirmish Shah, Paula Tanabe, Julie Kanter
Summary: This study aimed to identify barriers to sickle cell disease (SCD) care from the perspective of individuals with SCD in a multi-state sample. The results showed that lack of knowledge, inadequate caregiver support, limited provider knowledge, and transportation barriers were major obstacles to optimal SCD care. Strategies tailored towards reducing these barriers are necessary to improve the quality of SCD care.
Article
Oncology
Kerri E. Woodward, Yelena L. Johnson, Lindsey L. Cohen, Carlton Dampier, Soumitri Sil
Summary: This study found that families were categorized into Universal, Targeted, and Clinical risk groups based on psychosocial risk, with patients in the Targeted group reporting significantly higher pain frequency than those in the Universal group. The association between pain frequency and health care utilization significantly varied on the basis of psychosocial risk, with Clinical psychosocial risk strengthening the connection between pain frequency and health care utilization. Integrating psychosocial screening tools like the PAT into routine clinical care may help health care providers better support families in managing pediatric sickle cell disease.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Medicine, General & Internal
Adeel Ahmad, Diana Mihalca, Ben Stacey, Sayna Samaee, Dipal Mehta, Stephen Hibbs, Tanya Freeman, Basabi Chatterjee, Enamul Ali, Leo Cheng, Dimitris A. Tsitsikas
Summary: This study examined the dental health of sickle cell disease patients and found that a majority of them required dental extractions. More than half of the patients did not have regular dental check-ups. The lack of correlation with disease severity suggests that poor awareness of the importance of dental health may be the main factor contributing to the high prevalence of severe dental disease.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Pharmacology & Pharmacy
Tim Preijers, Lisette M. Schutte, Marieke J. H. A. Kruip, Marjon H. Cnossen, Frank W. G. Leebeek, Reinier M. van Hest, Ron A. A. Mathot
Summary: Hemophilia A and B are bleeding disorders caused by deficiencies of clotting factor VIII and IX. Treatment involves prophylactic administration of clotting factor concentrates or desmopressin to prevent bleeding. Population pharmacokinetic analyses play a crucial role in identifying patient characteristics and optimizing individualized dosing strategies.
CLINICAL PHARMACOKINETICS
(2021)
Review
Hematology
J. J. Gerritsma, I. Oomen, S. Meinderts, C. E. van der Schoot, B. J. Biemond, J. G. van der Bom, K. Fijnvandraat
Summary: RBC alloimmunization is a serious complication of blood transfusions, with genetic factors potentially influencing the risk. High-quality evidence on genetic risk factors for alloimmunization is urgently needed as current evidence is of moderate to low quality. The alleles HLA-DRB1*04, HLA-DRB1*15, and HLA-DRB1*03 showed significant associations with alloimmunization to the Duffya antigen, while HLA-DRB1*10, HLA-DRB1*11, and HLA-DRB1*13 were associated with anti-K formation.
Article
Oncology
Djamila E. Issa, Avinash G. Dinmohamed, Marielle J. Wondergem, Hedwig M. Blommestein, Peter C. Huijgens, Pieternella J. Lugtenburg, Otto Visser, Sonja Zweegman, Martine E. D. Chamuleau
Summary: Randomized controlled trials have confirmed similar efficacy of different dose-intensity and dose-interval regimens of R-CHOP in DLBCL patients across different age groups, while overall survival decreased with older age. Interestingly, in patients aged 18-64, the adjusted risk of mortality with 6xR-CHOP21 compared to other R-CHOP regimens appears to be similar.
LEUKEMIA & LYMPHOMA
(2021)
Article
Health Care Sciences & Services
Oriana Ciani, Bogdan Grigore, Hedwig Blommestein, Saskia de Groot, Meilin Mollenkamp, Stefan Rabbe, Rita Daubner-Bendes, Rod S. Taylor
Summary: This study mapped methodologies for the validation of surrogate endpoints and determined their impact on the acceptability of surrogates and coverage decisions made by HTA agencies. Results showed varying handling of surrogate endpoint evidence across agencies, with inconsistent consideration of the level of evidence and statistical validation in HTA reports.
MEDICAL DECISION MAKING
(2021)
Article
Economics
Stefan Rabbe, Meilin Moellenkamp, Benedetta Pongiglione, Hedwig Blommestein, Pim Wetzelaer, Renaud Heine, Jonas Schreyoegg
Summary: Variation in healthcare utilization is mainly attributed to hospital level factors, with regional characteristics explaining only a small part of the variation. Effective care category shows less variation compared to preference- and supply-sensitive categories.
Review
Oncology
Yi Hsuan Chen, Dominique Molenaar, Carin A. Uyl-de Groot, Marco van Vulpen, Hedwig M. Blommestein
Summary: This study summarized evidence of healthcare resource use and costs related to radiotherapy-induced adverse effects and found significant differences in economic burden estimation due to variations in study design, population characteristics, cost estimation methodologies, and healthcare systems across different countries.
Editorial Material
Hematology
Hedwig M. Blommestein, Sonja Zweegman
Summary: In this study, the first-line treatment with daratumumab in combination with different drugs was found to increase quality adjusted life years (QALYs) and reduce treatment costs compared to delaying daratumumab treatment and using it as a second-line therapy with carfilzomib and dexamethasone.
Article
Cell & Tissue Engineering
Frederick W. Thielen, Renaud J. S. D. Heine, Sibren van den Berg, Renske M. T. ten Ham, Carin A. Uyl-de Groot
Summary: Drug prices play a significant role in the accessibility and affordability of novel therapies. This study used a cost-based pricing model to calculate the prices of cell and gene therapies, with OTL-200 and AVXS-101 as case study examples.
Article
Economics
Ngoc Do, Frederick W. Thielen
Summary: This study aimed to evaluate the cost-effectiveness of VenO compared with ClbO in the treatment of treatment-naive adult patients with CLL from a Dutch societal perspective. The results showed that VenO was dominant over ClbO in treating CLL in treatment-naive adult patients assuming a Dutch societal perspective.
Article
Health Policy & Services
Christine Bennink, Marjolein van der Klift, Pieter Sonneveld, Jan A. Hazelzet, Hedwig M. Blommestein
Summary: This study compared the treatment burden in patients with Multiple Myeloma (MM) and Chronic Lymphoid Leukaemia (CLL) using retrospective analysis and Electronic Health Records (EHR) data. The findings showed that MM patients generally have a higher treatment burden, especially in the first year after diagnosis.
HEALTH POLICY AND TECHNOLOGY
(2022)
Article
Oncology
Yi Hsuan Chen, Hedwig M. Blommestein, Reinder Klazenga, Carin Uyl-de Groot, Marco van Vulpen
Summary: Proton therapy delivers more precise treatment compared with conventional radiotherapy. However, the limited information about the treatment costs per patient might affect decision-making. This study aims to calculate the costs of Proton therapy at a single center during the start-up phase and provide essential information for health technology assessment.
Article
Hematology
Brenda Leeneman, Hedwig M. Blommestein, Annemieke van Dongen-Leunis, Mattia Algeri, Willem E. Fibbe, Liesbeth Oosten, Carin A. Uyl-de Groot, Frederick W. Thielen
Summary: This study found that patients with steroid-refractory acute graft-versus-host disease (SR-aGvHD) have poor health-related quality of life (HRQoL). Patients face significant problems in areas such as usual activities, pain/discomfort, mobility, self-care, and anxiety/depression. Improving HRQoL and symptom management should be prioritized for these patients.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)
Article
Oncology
Marianne Luyendijk, Otto Visser, Hedwig M. Blommestein, Ignace H. J. T. de Hingh, Frank J. P. Hoebers, Agnes Jager, Gabe S. Sonke, Elisabeth G. E. de Vries, Carin A. Uyl-de Groot, Sabine Siesling
Summary: This study evaluated the changes in survival of patients with de novo metastatic solid cancers over the past 30 years. The proportion of M1 disease and net survival rates varied among different cancer types. Better preventive measures and early detection are needed to reduce the incidence of metastatic disease.
JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE
(2023)
Article
Hematology
M. R. Seefat, D. G. J. Cucchi, K. Groen, M. L. Donker, K. G. van Der Hem, M. Westerman, A. M. Gerrits, A. Beeker, N. W. C. J. van de Donk, H. M. Blommestein, S. Zweegman
Summary: Novel therapies for multiple myeloma have improved survival rates, but their high costs strain healthcare budgets. This study analyzed the treatment sequences and drug costs of MM patients in the Netherlands who died between January 2017 and July 2019. The study found that end-of-life treatment costs are substantial despite limited survival benefits, suggesting a need for further research and cost management.
EUROPEAN JOURNAL OF HAEMATOLOGY
(2023)