scholarly journals SEVERE COMBINED IMMUNODEFICIENCY SCREENING AND STEM CELL TRANSPLANT-AN ECONOMIC EVALUATION SCHEME

2021 ◽  
Vol 9 (11) ◽  
pp. 966-969
Author(s):  
Husain Ahmed Alzobaidi ◽  
◽  
Abdul Rhman Alghamdi ◽  
Mohammad Eid Mahfouz ◽  
Rawan Dakeel Allah Alsubhi ◽  
...  
Keyword(s):  
Stem Cell ◽  
Economic Evaluation ◽  
Stem Cell Transplant ◽  
Severe Combined Immunodeficiency ◽  
Cost Effective ◽  
Standard Of Care ◽  
Cell Transplant ◽  
Combined Immunodeficiency ◽  
Current Medical Practice ◽  
The Cost

Background: Severe Combined Immunodeficiency (SCID) is a rare genetic disease that affects newborn immune system and costs healthcare system a substantial amount of money. Screening and stem cell transplant may offer a cost-effective alternative to the current medical practice. Objective: To determine the cost-effectiveness of screening newborn for SCID with subsequent stem cell transplant versus standard of care. Materials and Methods: An economic evaluation was proposed, using Treeage software on a set of evidence-based propabilities and variables. Outcomes: as shown on the tree, we count the death from SCID. Its given the payoff 1, while the survival is payoff 0. The outcome of interest is death averted by screening live birth and implementing subsequent stem cell transplant for those who tested +ve.

scholarly journals A Decade Experience on Severe Combined Immunodeficiency Phenotype in Oman, Bridging to Newborn Screening

2021 ◽  
Vol 11 ◽  
Author(s):  
Nashat Al Sukaiti ◽  
Khwater Ahmed ◽  
Jalila Alshekaili ◽  
Mahmood Al Kindi ◽  
Matthew C. Cook ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Severe Combined Immunodeficiency ◽  
Diagnostic Delay ◽  
Cell Transplant ◽  
Combined Immunodeficiency ◽  
Hematopoietic Stem ◽  
Tertiary Center

IntroductionSevere combined immunodeficiency (SCID) results from various monogenic defects that impair immune function and brings on early severe and life-threatening infections. The main stay of treatment for SCID is hematopoietic stem cell transplant (HSCT) with near normal survival at 5 years for an early transplant done at or before the age of 3.5 months of life and the patient is maintained free of infections. Although overall rare, it constitutes a major burden on affected children, their families and on the health system especially in communities with a high rate of consanguinity where incidence and prevalence of recessive inborn errors of immunity (IEI) are expected to be high.MethodHere, we report the clinical, immunological, and molecular findings in 36 children diagnosed with SCID from a single tertiary center in Oman for the last decade.ResultsWe observed a median annual incidence rate of 4.5 per 100,000 Omani live births, and 91.7% of affected children were born to consanguineous parents. Twenty-three children (63.9%) fulfilled the criteria for typical SCID. The median age at onset, diagnosis and diagnostic delay were 54, 135, and 68 days, respectively. The most common clinical manifestations were pneumonia, septicemia, and chronic diarrhea. Eleven children (30.6%) have received hematopoietic stem cell transplant (HSCT) with a survival rate of 73%. The most frequent genetic cause of SCID in this cohort (n = 36) was (RAG-1), encoding for recombination activating gene (n = 5, 13.9%). Similarly, Major histocompatibility complex type II deficiency accounted for (n = 5, 13.9%) of our cohort.ConclusionOur report broadens the knowledge of clinical and molecular manifestations in children with SCID in the region and highlights the need to initiate newborn based screening program (NBS) program.

scholarly journals A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK

2019 ◽  
Vol 5 (3) ◽  
pp. 28 ◽  
Author(s):  
Alice Bessey ◽  
James Chilcott ◽  
Joanna Leaviss ◽  
Carmen de la Cruz ◽  
Ruth Wong
Keyword(s):  
Cost Effectiveness ◽  
Severe Combined Immunodeficiency ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Combined Immunodeficiency ◽  
Tree Model ◽  
Life Years ◽  
The Uk ◽  
The Cost ◽  
The Impact

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built. Model structure and parameterisation were informed by systematic review and expert clinical judgment. A public service perspective was used and lifetime costs and quality-adjusted life years (QALYs) were discounted at 3.5%. Probabilistic, one-way sensitivity analyses and an exploratory disbenefit analysis for the identification of non-SCID patients were conducted. Screening for SCID was estimated to result in an incremental cost-effectiveness ratio (ICER) of £18,222 with a reduction in SCID mortality from 8.1 (5–12) to 1.7 (0.6–4.0) cases per year of screening. Results were sensitive to a number of parameters, including the cost of the screening test, the incidence of SCID and the disbenefit to the healthy at birth and false-positive cases. Screening for SCID is likely to be cost-effective at £20,000 per QALY, key uncertainties relate to the impact on false positives and the impact on the identification of children with non-SCID T Cell lymphopenia.

scholarly journals Treatment dilemma for survivors of rituximab-induced bowel perforation in the setting of post-transplant lymphoproliferative disorder

2018 ◽  
Vol 11 (1) ◽  
pp. e226666 ◽  
Author(s):  
Brianne J Sullivan ◽  
Grace J Kim ◽  
Gabriel Sara
Keyword(s):  
Monoclonal Antibody ◽  
Stem Cell ◽  
Lymphoproliferative Disorder ◽  
Stem Cell Transplant ◽  
Bowel Perforation ◽  
Standard Of Care ◽  
Haematopoietic Stem Cell ◽  
Cell Transplant ◽  
Tumour Lysis ◽  
Post Transplant

Post-transplant lymphoproliferative disorder (PTLD) is a recognised complication of solid and haematopoietic stem cell transplant. It consists of a heterogeneous group of lymphoid neoplasms that arises secondary to post-transplant immunosuppression. Although there is no definite standard of care for the optimal treatment for PTLD, rituximab, a monoclonal antibody, with and/or without chemotherapy (usually CHOP=cytoxan, doxorubicin, vincristine, prednisone) has become a routine part of the treatment of any CD20 (+) PTLD, with response rates similar to chemotherapy with decreased toxicity. A rare and often lethal, complication of rituximab therapy for PTLD is bowel perforation secondary to tumour lysis of lymphoma involving the intestine. A small number of cases of bowel perforation have been reported, with very few documented survivors. The risk for recurrent perforation in the setting of ongoing rituximab treatment is unknown. There is sparse data supporting how to best treat the survivors.

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