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1.
Hematol., Transfus. Cell Ther. (Impr.) ; 43(4): 476-481, Oct.-Dec. 2021. tab, ilus
Article in English | LILACS | ID: biblio-1350816

ABSTRACT

ABSTRACT Introduction: We performed cost-effectiveness and cost-utility analyses of the modified International Consortium on Acute Promyelocytic Leukemia protocol in Mexico for the treatment of acute promyelocytic leukemia Acute Promyelocytic Leukemia. Methods: We performed a three-state Markov analysis: stable disease (first line complete response [CR]), disease event (relapse, second line response and CR) and death. The modified IC-APL protocol is composed of three phases: induction, consolidation and maintenance. Cost and outcomes were used to calculate incremental cost-effectiveness ratios (ICERs); quality-adjusted life-years were used to calculate incremental cost-utility ratios (ICURs). Results: The CR was achieved in 18 patients (90%), treated with the IC-APL protocol as the first-line option; one patient (5%) died in induction, another one never achieved CR (5%); of the 18 patients that achieved CR, 1 relapsed (5.5%). The median treatment cost of the IC-APL protocol was $21,523 USD. The average life-year in our study was 7.8 years, while the average quality-adjusted life-year (QALY) was 6.1 years. When comparing the ICER between the IC-APL and the all-trans retinoic acid (ATRA) plus arsenic trioxide (ATO) protocols, we found the different costs of $6497, $19,133 and $17,123 USD in Italy, the USA and Canada, respectively. In relation to the ICUR, we found the different costs to be $13,955 and $11,979 USD in the USA and Canada, respectively. Conclusion: Taking into account the similar response rates, lower cost and easy access to the modified IC-APL regimen, we consider it a cost-effective and cost-utility protocol, deeming it the treatment of choice for our population.


Subject(s)
Humans , Male , Female , Adolescent , Adult , Middle Aged , Aged , Leukemia, Promyelocytic, Acute/diagnosis , Leukemia, Promyelocytic, Acute/drug therapy , Tretinoin/therapeutic use , Clinical Protocols , Cost-Benefit Analysis
2.
Arch. argent. pediatr ; 113(3): e137-e139, jun. 2015.
Article in Spanish | LILACS | ID: lil-750470

ABSTRACT

El síndrome de Wiskott-Aldrich es una inmunodeficiencia primaria; con una incidencia de 3,5 a 5,2 por cada millón de recién nacidos masculinos. Se caracteriza por tener un patrón de herencia recesiva ligada al cromosoma X. En estos pacientes; se ha descrito la tríada clásica de inmunodeficiencia; microtrombocitopenia y eczema. Presentamos un paciente de 5 años de edad; hispánico; con antecedentes de numerosas infecciones desde el primer año de vida. Actualmente; presenta desnutrición crónica; talla baja secundaria y retraso en el desarrollo del lenguaje. Se diagnosticó una mutación poco frecuente del gen asociado al síndrome de Wiskott-Aldrich.


The Wiskott-Aldrich syndrome is a rare X-linked recessive immunodeficiency, with an estimated incidence of 3.5 to 5.2 cases per million males. It is characterizedby immunodeficiency, microthrombocytopenia and eczema. We present a 5-year-old Hispanic male, with a medical history of numerous infectious diseases, compromised health, chronic malnutrition, language delay and failure to thrive. An infrequent mutation in the Wiskott-Aldrich syndrome gene was found.


Subject(s)
Animals , Chick Embryo , Avian Proteins/metabolism , Cadherins/metabolism , Neural Stem Cells/cytology , Neural Stem Cells/metabolism , Avian Proteins/antagonists & inhibitors , Avian Proteins/genetics , Base Sequence , Cell Count , Cadherins/antagonists & inhibitors , Cadherins/genetics , Gene Expression Regulation, Developmental , Gene Knockdown Techniques , Neural Tube/cytology , Neural Tube/embryology , Neural Tube/metabolism , Oligonucleotide Array Sequence Analysis , Phenotype , RNA Interference , RNA, Small Interfering/genetics , Signal Transduction
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