scholarly journals Targeted Busulfan therapy with a steady-state concentration of 600–700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant

2013 ◽  
Vol 49 (3) ◽  
pp. 366-369 ◽  
Author(s):  
S Maheshwari ◽  
A Kassim ◽  
R F Yeh ◽  
J Domm ◽  
C Calder ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Steady State ◽  
Sickle Cell Disease ◽  
Bone Marrow Transplant ◽  
Sickle Cell ◽  
Marrow Transplant ◽  
Steady State Concentration ◽  
Cell Disease ◽  
State Concentration

scholarly journals Comparative Cost Analysis of Therapy in Sickle Cell Anemia: Supportive Care Vs. Bone Marrow Transplant

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4466-4466 ◽  
Author(s):  
Pooja Lothe ◽  
Tiffany Pompa ◽  
Maneesh Jain ◽  
Parshva Patel ◽  
Yayu Liang ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Sickle Cell Disease ◽  
Length Of Stay ◽  
Bone Marrow Transplant ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Hospital Admissions ◽  
Marrow Transplant ◽  
Cell Disease ◽  
The Cost

Abstract Objective: A comparative cost analysis of sickle cell admissions vs. stem cell transplants in sickle cell patients. Hypothesis: We believe the overall cost of a bone marrow transplant for a sickle cell patient will be less than that of a patient with multiple sickle cell admissions. Background: Sickle cell disease remains an increasing burden to the cost of health care and health care providers. The disease results in a variety of serious organ system complications that can lead to life-long disabilities and/or early death. Despite the advent of hydroxyurea, sickle cell admissions and cost have been increasing over the course of several years. Various contributing factors may include socioeconomic status, complications of sickle cell anemia itself, narcotic dependence and noncompliance with medications. Bone marrow transplants were introduced in 1982 as an option for the treatment of sickle cell anemia, and are currently the only curative option in this disease. A study conducted at the NIH from 2004-2013, found that bone marrow transplant reversed the disease in 26 of 30 patients (87%) (Hseih et al, N Engl J Med 2009; 361:2309-2317). The patients ultimately had a normal hemoglobin, fewer hospitalizations, and lower use of narcotics to treat pain from the disease. However, the underutilization of bone marrow transplant continues to exist and may in part be secondary to a lack of fully matched donors. To overcome this challenge, the Johns Hopkins group developed a nonmyeloablative bone marrow transplantation platform using HLA haploidentical donors for patients using posttransplant cyclophosphamide. As a result, 17 patients were successfully transplanted, 14 from HLA-haploidentical and 3 from HLA-matched related donors (Meade et al, Blood. 2012; 120(22):4285-4291). Due to this, most patients with sickle cell disease have the potential to undergo a successful bone marrow transplant. However, an analysis comparing the cost of admissions vs. transplant has yet to be determined. In order to create an effective cost comparison, we utilized a nationwide database. Methods: US hospital admissions were identified from discharge data from the Nationwide Inpatient Sample (NIS), Healthcare Cost and Utilization Project (HCUP), Agency for Healthcare Research and Quality from 1998 to 2011 using ICD9 codes. Admissions were included if they had an ICD9 code for Sickle cell anemia (282.5). Results: Table 1. Year 1998 1999 2000 2001 2002 2003 2004 Length of Stay (Days) 6.21 6.13 6.37 6.19 6.46 6.31 6.05 Cost ($) 15,724.00 16,465.00 18,654.00 18,750.00 22,587.00 24,501.00 23,743.00 Table 2. Year 2005 2006 2007 2008 2009 2010 2011 Average Length of stay (Days) 6.16 6.13 6.00 5.91 5.62 5.71 5.61 6.04 Cost ($) 25,129.00 27,471.00 28,425.00 27,314.00 29,767.00 29,929.00 31,683.00 24,687.00 The cost per hospitalization has almost doubled since 1998, despite a slight decrease in length of stay from an average of 6.5 days to 5.6 days from 1998-2011. According to the data base from our study, the average cost per patient per hospitalization was $24,687. The average number of admissions per year for a single patient with sickle cell anemia is 6 (Ballas et al, Am J Hem 2009) for an estimated overall cost per year of $148,000. However, this value underestimates the true cost since this does not include emergency room visits, medicine costs, and readmission rates. Conclusion: In the age of cost effective medicine, clinicians struggle to find a balance between low cost and optimal patient care. An underutilized modality of care and even cure for sickle cell disease is bone marrow transplant. The sickle cell information center website estimates the cost of the transplant process for most patients to be $150,000 to $250,000 which includes pre-transplant evaluation, transplant stay, and post-transplant follow-up (https://scinfo.org). We estimate that a bone marrow transplant is approximately equivalent to the cost of ten sickle cell hospital admissions. According to this analysis, undergoing a bone marrow transplant would ultimately prove to be more cost efficient while decreasing the rate of complications associated with this debilitating disease. Disclosures No relevant conflicts of interest to declare.

Successful bone marrow transplant for sickle cell disease after non-myeloablative preparation

2000 ◽  
Vol 22 (4) ◽  
pp. 387
Author(s):  
A Woolfrey ◽  
C Hoppe ◽  
R A Nash ◽  
J E Sanders ◽  
E Vichinsky ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Sickle Cell Disease ◽  
Bone Marrow Transplant ◽  
Sickle Cell ◽  
Marrow Transplant ◽  
Cell Disease

scholarly journals Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease

2020 ◽  
Vol 3 (5) ◽  
pp. e206742 ◽  
Author(s):  
Nitya Bakshi ◽  
Deeksha Katoch ◽  
Cynthia B. Sinha ◽  
Diana Ross ◽  
Maa-Ohui Quarmyne ◽  
...  
Keyword(s):  
Decision Making ◽  
Bone Marrow ◽  
Sickle Cell Disease ◽  
Bone Marrow Transplant ◽  
Sickle Cell ◽  
Marrow Transplant ◽  
Cell Disease

scholarly journals Experiences and Decision Making for Bone Marrow Transplant in Sickle Cell Disease: Patients and Caregivers Perspective

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2393-2393
Author(s):  
Kirshma Khemani ◽  
Diana Ross ◽  
Ann Haight ◽  
Cynthia Sinha ◽  
Nitya Bakshi ◽  
...  
Keyword(s):  
Decision Making ◽  
Bone Marrow ◽  
Sickle Cell Disease ◽  
Bone Marrow Transplant ◽  
Sickle Cell ◽  
Treatment Option ◽  
Marrow Transplant ◽  
Cell Disease ◽  
Entire Family ◽  
Age Range

Abstract Background:Bone marrow transplantation (BMT) is currently the only curative treatment option available for patients with sickle cell disease (SCD). There are very limited data available on the patients and caregivers perspective on decision making for BMT and their overall experiences with the BMT process. The objective of this study was to determine patients and caregivers knowledge of BMT, the factors that influenced families to consider BMT as a treatment option and their experiences during and after transplantation. Methods:At Children's Healthcare of Atlanta, we conducted 22 qualitative interviews involving patient-caregiver dyads using a semi-structured interview guide. The questions focused on patient and caregiver decision-making and their experience with bone marrow transplant for SCD. All interviews were recorded and transcribed verbatim. Transcripts were then double-coded. Content analysis was performed using NVivo 10. Results: Eleven patients (n=6; Females) and 11 caregivers (n=10, Mothers) participated. At the time of interview, patient's age range was 11-32 years with a median value of 20 years; at the time of transplant, patient's age range was 6 to 17 years with a median value of 11 years. Time from transplant ranged from 0-17 years with a median value of 2 years. Analysis revealed that most patients' and caregivers' were presented with BMT as a treatment option based on the severity of patient's clinical situations. Six participants, patients and caregivers combined, described minimal knowledge and understanding of the BMT process and/or the associated risks/benefits involved with transplant before consultation with the BMT team. Both patients and caregivers reported that the decision to move forward with BMT was strongly influenced by the progression of sickle cell related complications. All patients were either suffering from chronic pain or had been on chronic transfusions for several years. Given the effects of BMT on the entire family, 7 patients and 5 caregivers reported the need to involve the entire family in the decision-making process. Other factors that played a significant role in the decision-making process for patients and caregivers included: information and education received from the BMT team, having an HLA-matched sibling or unrelated donor, and spirituality. A few patients and caregivers stated that hearing from other families would have provided them a more realistic understanding of the transplant process and further aided in their decision-making. Of the 7 patients and caregivers asked about fertility issues and its impact on transplant decision making, 3 patients, and 3 caregivers conveyed that it was the "least of their concerns" and did not feel the need to proceed with fertility preservation. One caregiver voiced concerns about fertility issues, but after consultation with a fertility specialist decided to proceed with BMT. About 95% of patients and caregivers described transplant day as a "re-birth day" or "new birthday". Most patients conveyed that having to deal with chemotherapy-related side effects, such as hair loss, the need for nasogastric tubes, and being isolated from family and friends was all "worth it" because they no longer have sickle cell disease. For 8 caregivers, prayers and family support played a prominent role in helping to cope with the fears and stress involved with BMT. Eight patients indicated that prayers and distractions such as watching movies, playing video games and doing school work helped with coping. Despite having to deal with the arduous transplant process and transplant-related complications, patients and caregivers alike were satisfied with their decision and expressed no decisional regret.While this study included some patients who had severe complications including chronic GVHD, we did not interview caregivers of any patient who had died following BMT. Conclusion: Decision making for bone marrow transplant as a treatment option for those with sickle cell disease is a complex process. Most patients and caregivers have limited knowledge about bone marrow transplant being a curative treatment for sickle cell disease, and the need to educate patients and families is crucial. Gaining an understanding of factors that influence decision making for this population and their experiences with the transplant process will generate crucial insights that will impact the care of future patients and research studies. Disclosures No relevant conflicts of interest to declare.

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