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[After disappointment regarding secondary cardiac prevention. What is the status of hormone replacement therapy today?].

ERT (estrogen replacement therapy) or HRT (hormone replacement therapy) can be employed for the prevention/treatment of various hormone-related conditions. The main indications are the treatment of psychovegetative deficits and--in part--primary prevention of osteoporosis. For other (hormone-related) diseases such as certain malignant tumors, the risk may increase under replacement treatment, so that a risk/benefit evaluation should always precede it prescription. Side effects of ERT/HRT include a greater risk of thrombosis and, possibly, a increase in cardiovascular events, so that this form of treatment cannot be recommended at least for the secondary prevention of cardiovascular disease. In general, treatment must be matched to the requirements and wishes of the patient. With increasing age and remitting symptoms, the dose can be reduced. Alternative substances such as phytoestrogens or selective estrogen receptor modulators (SERMs) extend the spectrum of therapeutic options.

Adult↗

FPA disappointed in Great Britain.

The Family Planning Association of Great Britain has released a statement on the role of family planning within the reorganized system of the National Health Service (NHS). The statement is critical of the limitations placed on availability of birth control means to NHS patients. It had been hoped that contraceptives would be free to all people covered by the NHS. The government, however, made the advice on family planning free to all, but the prescriptions and supplies would be given free only in cases of social or financial need or to women within 12 months after a birth or an abortion. Thus, the effect upon the decrease of the number of abortions and unplanned pregnancies will not be as great as it was hoped it would be if contraceptive means would be supplied freely.

Contraception↗

[Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments].

Children with severe combined immunodeficiency (SCID) die within 2 years of age if untreated. The only effective treatment for SCID since 1968 is a hematopoetic stem cells (HSC) transplantation. Only 25% of patients have an HLA matched related donor, while the rest have to be transplanted with T cells depleted haploidentical parental bone marrow, unrelated bone marrow or unrelated umbilical cord blood. In many cases, however, despite a positive outcome, children are not achieving B cell reconstitution and require regular IV Ig infusion. Gene therapy with genetically modified autologous cells offers a cure with no immunological complications such as graft rejection, graft versus host disease (GVHD) or post-transplantation immunosuppressive therapy. The first gene therapy trials were introduced in 1990 for adenosine deaminase (ADA) deficient patients who had failed to respond to PEG-ADA. Since then, three clinical trials have evaluated the transplantation of ex-vivo transduced autologous haematopoietic stem cells (HSC) to treat ADA deficiency. One trial used only bone marrow HSC, a second used bone marrow plus peripheral blood T lymphocytes, and a third used umbilical cord blood HSC. These trials give promise but also define the present limitations of gene therapy. Future protocols might be adjusted according to the new observations that ADA-expressing T cells have a strong selective advantage over ADA-deficient T cells. PEG-ADA enzyme therapy might be therefore contraindicated. Another new strategy might involve moderate conditioning prior to the reinfusion of genetically modified CD34+ cells, "making space" for transplanted HSC. The first successful gene therapy was reported for treatment of X-linked severe combined immunodeficiency (SCID-X1) in Science 2000. Since then, the group at the Hopital Necker in Paris has treated 11 patients with ex-vivo gene therapy for the deficiency of the common g chain. All eleven boys are alive, however, one of them recently developed a leukaemia-like disease. This case is being investigated to determine whether the genetic manipulations of the patient's HSC could be the reason for mutagenesis and how other factors could have contributed to this unfortunate event.

Adenosine Deaminase↗