Of 307 patients followed by our Center at the time of the survey, 159 patients were identified as eligible for our study. Of these, 100 of their caregivers agreed to participate and a total of 100 patients were analyzed. Table 1 reports their baseline characteristics. The median age was 12 (IQR 5–16). The majority of patients were of Italian origin (n = 73, 73%). 93% have been followed for a congenital or perinatal-onset condition and only two patients had an oncological condition. The most common primary diagnostic categories were neurologic (n = 55, 55%) and musculoskeletal (n = 23, 23%). A total of 26 patients had the DNR order and three patients were in a terminal stage of their disease. Due to medical conditions and/or their age, 85 individuals could not self-feed and 62 of them had feeding tubes such as nasogastric (NG), gastrostomy (G) or jejunostomy (J) tubes.

Figure 1 (TITLE: Prevalence of principal symptoms and needs according to different body systems) represents principal symptoms and needs prevalence across the population. Neurological, gastrointestinal and respiratory symptoms were the most prevalent. The most common symptoms reported were: cognitive impairment (n = 74, 74%), mobility impairment (n = 55, 55%) and seizures (n = 53, 53%) for neurological symptoms, dysphagia (n = 79, 79%), inadequate oral intake (n = 56, 56%) and constipation (n = 44, 44%) for gastrointestinal symptoms and excessive respiratory secretions (n = 46, 46%) and chronic respiratory failure (n = 45, 45%) for respiratory symptoms. We reported also 7 individuals with sexual needs. (See the table in additional file 2 for a detailed report of symptoms and needs.)

Prevalence of principal symptoms and needs according to different body systems

Table 2 describes caregivers’ characteristics and their social-cultural aspects. 95% of patients had natural parents. The main caregiver was the mother in 91% of cases. No significant difference in education degree was found between fathers and mothers. However, mothers had a significantly higher unemployment rate. The average number of cohabitants in the family resulted in 4 ± 1.4 (median 4, IQR 3–5). 69% of patients had siblings, of these 43% were younger and 26% older.

Table 3 shows the prevalence of polypharmacy and details of patients’ therapies. 67% of patients were on polypharmacy defined as the current assumption of five or more different drugs. The median number of drugs prescribed for patients was 7 (IQR 4–11). The median number of total administrations per day was 10 (IQR 4–16), with a maximum of 35. Each treatment regimen included a median of 3 (IQR 2–4) ATC classes. A total of 741 drug prescriptions were analyzed. The most prevalent drug classes were represented by neurologic (n = 286, 38.6%), alimentary tract and metabolism (n = 251, 33.9%) and respiratory (n = 38, 5.1%). See Fig. 2 for more detail (TITLE: Prevalence of different drugs according to ATC classification). Oral and enteral assumptions were the most frequent routes of administration (n = 679, 91.6%). Other routes of administration as sublingual, rectal, transcutaneous, inhalation and intranasal account together for 55 prescriptions (7.5%) and parenteral administrations (intravenous, intramuscular) only 7 prescriptions (0.9%).

Prevalence of different drugs according to ATC classification; Legend “ATC: Anatomical Therapeutic Chemical (ATC) classification. Total drugs, n = 741. N: Nervous system (n = 286); A: Alimentary tract and metabolism (n = 251); R: Respiratory system (n = 38); J: Anti-infective for systemic use (n = 33); B: Blood and blood forming organs (n = 32); M: Musculoskeletal system (n = 32); C: Cardiovascular system (n = 29); H: Systemic hormonal preparations, excluding sex hormones and insulins (n = 16); S: Sensory organs (n = 9); L: Antineoplastic and immunomodulating agents (n = 7); D: Dermatological (n = 6); G: Genito urinary system and sex hormones (n = 2)”

10% of respondents reported difficulties in preparing therapies and 15% encountered challenges in accurately measuring the medication dose. The time required for therapy preparation varied among respondents, with 68% spending less than five minutes, 19% between 5 and 10 min, and 13% more than 10 min. Medication handling (tablet crushing, capsule opening or dilution and administration via feeding tubes) was widespread among responders (87%) and 52% of them needed to handle it at least 3 times per day. 59% of responders felt adequately trained for therapy preparation. 65% received training from our PPC center healthcare providers, 17% from the hospital healthcare providers, 12% from their pediatrician or general practitioner, 4% from their neurologist and one from a pharmacist.

43% percent of the caregivers reported difficulties in adhering to the time intervals between administrations. Oral and enteral have been reported as the preferred route of administration and they were considered well tolerated and more adapted to the patients’ clinical conditions. 75% of caregivers reported no difficulties in the child’s acceptance of the administered therapy. All cares of children with a feeding tube reported no difficulties or “rarely”. 43% mixed medications with soft foods or beverages (e.g. milk, fruit juices, sweetened drinks), to facilitate administration and to improve acceptance. 55% of them did it for each administration.

14% of caregivers reported at least an administration error in the previous 3 months. The tiredness (35.7%) and the complexity of the therapeutic regimens (28.6%) were reported as the principal causes. See Table 4.

77% were satisfied with the current medication regime and the symptoms control achievement, although 41% thought their child takes too many medications “always” or “often”. 39% reported they wanted to discuss with medical staff their doubt about therapies. Lastly, 73% responded positively to the possibility of discussing the patient’s therapy with a clinical pharmacist.

The whole survey responses are provided as additional file 3.

Figure 3 (TITLE: Correlation analysis between the SCARES score and the error risk) illustrates the correlation analysis between the SCARES score and the error risk. As depicted in the chart, despite being able to ascertain the score for only 62 caregivers, the correlation is positive with r = 0.53 (p < 0.05). The main represented items were: “need to manipulate the drug before administration” (52/62), “patient with polypharmacotherapy” (47/62) and “need for night-time administration” (42/62).

Correlation analysis between the SCARES score and the error risk