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2015, Number 4

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Rev Hematol Mex 2015; 16 (4)

15-year experience in a medical center of care of patients with paroxysmal nocturnal hemoglobinuria

Alvarado-Ibarra M, De la Peña-Celaya A, López-Hernández M

Language: Spanish
References: 21
Page: 239-248
PDF size: 359.18 Kb.


ABSTRACT

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an orphan disease that affects deeply the quality of life and productivity of the patients. Our unit, on having treated itself about a medical center of the third level, has the aptitude to diagnose and treat these patients; nevertheless we do not possess casuistry of these patients.
Objective: To know the clinical, biochemical and epidemiological characteristics, as well as the evolution, therapeutic interventions and the response of patients with PNH attended in the course of 15 years in Service of Haematology of the Medical National Center 20 de Noviembre, Mexico City.
Patients and method: A retrospective, descriptive, longitudinal study was done with patients joined the period from January 1st, 2000 to December 31st, 2014, the variables analyzed were: age, sex, hemoglobin and number of transfusions, PNH variety, treatment and response. Diagnose was made with Ham’s positive test, positive Saccharose or FLAER, all the patients get through bone marrow aspiration. The information was obtained of the clinical and electronic records and own sheets of compilation of the Service. All the variables were analyzed in the software SPSS 21.
Results: During the period of study there were received in the Service 2,551 first time patients, managing to fulfill criteria for PNH in 8 cases, with an average age of 38 years, distributed in 38% women and 62% men. To the moment of their revenue it was possible to classify to 63% as PNH type III clone and 37% PNH type II clone. 50% of them received treatment with eculizumab and the rest, with danazol, steroid and/or measures of support. Only one patient presented with thrombosis. The average of initial hematocrit of the patients who received eculizumab was 23% and later to the treatment of 31%, and those who received another treatment was 32% and it kept equal. The basal DHL of the patients who received eculizumab was of 2,960 mg/L and the later one of 298 mg/dL and in other treatment group of 690 mg/dL and 1,545 mg/dL, respectively. The average of basal reticulocyte of the patients with eculizumab was 13.5% and later to treatment 4%, those of another treatment were 3% and 4%. The average of red blood cells transfused per month for the patients treated with eculizumab was of 3 and later to treatment it was of 0.75, for the agreements in another treatment group it was of 0.5 and later to treatment they kept equal. A patient who received eculizumab died due to complications not related to the disease or the treatment.
Conclusion: Patients who received eculizumab were diagnosed with type III clone and demonstrated improvement in the evaluated parameters and quality of life, the rest of patients were PNH type II clone and the current results show increase of reticulocytes and DHL still without repercussion in the number of hematocrit and transfusional therapy.


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