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Background Imatinib (Gleevec) is a protein kinase inhibitor initially registered in Italy as an orphan drug for the treatment of chronic myeloid leukaemia (CML) and gastro-intestinal stromal tumours (GIST): its use in clinical practice began in 2004 with a cost to the public per pack of 3,313.47 € (2,007.67 € ex -factory). The dosage varies from 400–800 mg/day and must be continued chronically until disease progression (DP) or until side effects appear. Purpose To examine the level of spending on imatinib in the National Cancer Institute of Milan (INT) compared with consumption in Italy. Materials and methods We interrogated the national reports on the use of drugs in Italy (OsMed) published by the AIFA requesting a report on consumption (extracted from the flow of File F for active drug) by number of treated patients and by department (SC Haematology and SC Sarcomas), in order to proceed to the comparison. Results Analysis of the data revealed that in the course of 5 years (2008–12) the expenditure at the national level went from 144,700,000 € to 122,478,260 € (period Jan–Sept 2012), peaking at 173,300,000 in 2011 €. In INT the figures were 2,419,605 € in 2008 to 2,881,185 € in 2012, peaking at nearly 3 million euro in 2011. The decrease since 2011 is considered to be linked to two factors: some patients showed resistance to the treatment, so they switched to use a second line treatment with sunitinib; some local health authorities have intervened in the supply of the drug to their patients. Conclusions In the period under consideration, INT expenditure was about 6% of the entire national expenditure, about 11.6% of the expenditure of the BPE. According to the latest epidemiological data, a slight increase is expected in new diagnoses with a consequent increase in spending both nationally and in individual structures. Will the NHS manage to ensure the continuity of care for life, with an average cost for each treatment of about 80 €/day? Is it necessary to wait for 2016 for the generic drug or should we turn to the Indian market, where the average cost is about 9 €/day? No conflict of interest.

In this paper the authors aim to assess the results of psychomotor treatment of 5 patients with cranial injuries admitted to the Division of Infantile Neuropsychiatry at Gaslini Hospital from 1983 to 1985 and to evaluate the role of this treatment in a pathology with multiple, complex functional disorders. Evolution was excellent in 3 out of 5 patients who presented level 1-3 coma with limited post-traumatic sequelae (epilepsy and dysfunction of the left side in A.D., epilepsy and learning disorders in C.F., motor impairment and learning disorders in V.D.) Effective control was achieved in epileptic patients together with a good recovery in those with learning handicaps. Only slight fine motor disorders remained in the case (A.D.) with left hemiparesis. The two cases with level 5 coma that developed into apallic syndrome both attained full postural and motor autonomy, although a slight paretic deficit remained in the left side in one case (V.A.) and vague cerebellar signs in the other (G.D.). Moreover, despite continuing residual difficulties involving concentration and instability of attention, together with slight affective and emotional problems, both girls acquired satisfactory knowledge and responsibility regarding their own psychomotor status and achieved good personal autonomy and a stable ego structure. These results appear to confirm the hypothesis that psychomotor treatment is an effective method of achieving an adequate recomposition of the psychophysical and functional unit of body, damaged by injury.

Background The compassionate use of drugs in Italy is a way of using drugs available in foreign countries but not in Italy. They have the authorization for the same therapeutic purpose but are not available on the market, however they are in clinical trials (CTs) or have been tested in phase III or – if the patient is in a critical condition – have been successful in phase II CTs. As regards evaluating the efficacy of the treatment, a basic aspect of the CT is to evaluate the safety of the drugs used. Great attention is focused on this theme with sponsored CTs; in fact by law (decree 211/2003) the subjects liable for pharmacovigilance are expressly listed and stress is put on reporting adverse drug reactions (ADRs). Regarding the use of compassionate drugs, there are no laws regulating the reporting of ADRs. Purpose To find out how any reports of possible ADRs emerging from compassionate studies are managed in ordinary clinical practise. Materials and Methods For this purpose the database used in our organisation was essential: all of our centre’s CTs are listed there. The number of CTs classified by type (for profit, not-for-profit, compassionate) was extracted, focusing on the compassionate use CTs in particular. Then the number of patients treated was drawn from it as well as the pathologies and the ADRs pointed out during meetings with the physicians. Results From this analysis it appeared that in our centre 197 studies (st) are active: 147 are for profit, 27 not-for-profit and 23 compassionate. Among these last ones, 8 are active in the sarcoma department, 5 in haematology, 3 in paediatrics, 6 in medical oncology and 1 in urology. The pathologies being examined are: GIST 4 active sts, 6 patients (pts), Hodgkin’s lymphoma 1 st (2 pts), T-cell lymphoma 1 st (1 pt), myeloma 2 sts (1 and 15 pts), thyroid carcinoma 1 st (1pt), bone metastases 1 st (4 pts), melanoma 2 sts (1 and 30 pts), villonodular tenosynovitis 1 st (4 pts), prostatic adenocarcinoma 1 st (4 pts), breast cancer 2 sts (3 and 2 pts), leiomyosarcoma 1 st (2 pts), myxoid liposarcoma 1 st (6 pts), brain stem glioma 1 st (8 pts), NET 1 st (17 pts), acoustic neuroma 1 st (1 pt), idiopathic myelofibrosis 1 st (1 pt), LLC 1 st (1 pt). Although the CTs for compassionate use are fewer than other kinds of trials and require a very low number of treated pts – also because it is a matter of a named patient use and in particular conditions – this does not justify not reporting ADRs. As such drugs are used for critical pts and often for non-approved uses, it seems useful to focus on this aspect, as it allows to more and better investigations on the side of the safety of the drugs. Conclusions The results obtained underline the necessity of better awareness of the problem. As far as our centre is concerned, the results led us to hold meetings with the physicians and to plan interventions in order to make them aware of the problem and in order to start a process of pharmacovigilance with compassionate-use drugs. No conflict of interest.

Background The Milan National Cancer Institute Pharmacy began a collaboration with the haematology and bone marrow transplantation (ETMO) department, to optimise concomitant conditioning protocols of bone marrow transplants; the pharmacokinetics and pharmacodynamics are affected by the high doses of chemotherapy administered. The drugs analysed were those in the conditioning schedules used in accordance with international guidelines. Purpose To provide a practical guide for managing drug interactions between the drugs commonly used by ETMO and those in the transplant conditioning schedules. Materials and Methods The presence of the ward pharmacist, funded by the Italian Haematology Society, allowed the daily management of treatment to be investigated. Databases were used (Micromedex, Codifa) and literature meta-analyses were conducted, in order to obtain the pharmacokinetic and pharmacodynamic characteristics of these drugs and possible interactions. Results Within our Institute, 72 transplants that used conditioning were performed in a year, 32 autologous and 40 allogeneic. In particular, 28 transplants used a high-dose melphalan scheme, 28 used thiotepa/fludarabine/cyclophosphamide, 4 used BEAM, 4 used TBI/fludarabine/cyclophosphamide and 8 used the KROGER scheme. Therefore the interactions between drugs used in the protocols themselves and the drugs commonly used within the department by transplant patients were analysed. For this purpose the following drugs were considered: ciclosporin, allopurinol, acetazolamide and IPP. Following this analysis, it was shown that there were significant interactions between the drugs used in the conditioning scheme and drugs commonly used in patients with bone marrow transplants. Conclusions The pharmacist set up a means of enabling a clinician to browse for a more informed choice: dedicated schemes are being developed, in which they report any interactions observed, associated with the treatment protocols. All this has therefore contributed to the rational use of the drugs and resources, for example the use of antifungals after transplantation and not before, and the introduction of pantoprazole instead of omeprazole. A future goal will be the analysis of the interactions between the drugs and concomitant haematology chemotherapy. No conflict of interest.

The aim of the work has been to verify the efficacy of a psychomotor rehabilitative treatment undertaken at a late age in two children with multiple handicaps: sensorial (visual and hearing impairment), motor (apostural-akinetic stage) and relational (autistic behavior).Salvatore C, and Ivan S., admitted to the "G. Gaslini" Institute after discharge from an Institution for homeless infants, were affected by prenatal rubella sequelae. They were taken in charge at age 11 and 24 months.manipulation and body-to-body interaction to reassure the children, relieve their feelings of fright and reduce the stereotyped behaviour; stimulation of the acquisition of the body-image; strengthening of the unimpaired sensorial skills and of the residual auditory and visual possibilities; postural and motor stimulation, following the autogenetic development; stimulation to achieve spatial organization and orientation; adjusting of interventional and relation modalities common to all the persons interacting with the children.we obtained a significant reduction of stereotyped behaviour and progressive improvement of the active research of productive interaction with the environment in both children; Salvatore C. was able to walk with little aid after one year of treatment; furthermore he reached a lively and organized motility, supported by a motivational drive. Ivan S. was able to walk with bilateral help after 4 months of treatment; the cognitive development improved at a slower pace; the child became able to reach for objects but used them only in stereotyped or elementary activities. From this observation it seems possible to confirm our hypothesis that psychomotor education is useful even if undertaken lately and in the absence of a supporting family.(ABSTRACT TRUNCATED AT 250 WORDS)