Protocols - Hemorio

CONCURRENT TREATMENTS
- Sodium alendronate 10mg/day, for patients with osteoporosis.
- The use of anticonvulsants is necessary for patients with type III.
- Polyvitamin mixtures, especially for children and elderly.
- Replacement with Vitamin B12, for the ones with anemia and low serum levels.
THERAPEUTIC PURPOSES
- Hb levels increased, maintained and sustained, in the first 12/24 months to ≥ 11g/dL in women and
children, and to ≥12 g/dL, in men.
- Platelet count increased in the first year of treatment, without spontaneous bleeding episodes and
without risk during surgical procedures.
- Reduction of liver volume in 20/30% maintained in the first year and 30/40% in 3 rd and 5 th year of
treatment.
- Reduction of spleen volume 30/50% in the first year and 50/60% in 2 nd and 5 th year of treatment,
resolving hypersplenism and discomfort associated to splenomegaly.
- Decrease or eliminate and prevent crisis of bone pain within 12/24 months and improve bone quality.
- Normalize the growth curve in children and delay normal signs of puberty in teenagers.
- Monitoring of pulmonary hypertension signs, reversion of hepatopulmonary syndrome and oxygen
dependence.
MULTI-DISCIPLINARY FOLLOW-UP & GENETIC ADVICE
Gaucher disease is multisystemic and therefore, it is recommended its evaluation with other specialists
whenever necessary. The specialists are: orthopedist, ophthalmologist, endocrinologist and cardiologist
are the ones most frequently requested. In HemoRio, there is a multi-disciplinary team following the
patients with specialized pharmaceutical assistance, evaluation with social and psychological assistance.
The children are followed along with pediatrics. And the geneticist appointment is also scheduled for
performance of genetic advice.
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