Cross-sectional analysis: clinical presentation of children with persistently low ALP levels.

OBJECTIVES: Low activity of serum alkaline phosphatase (ALP) is a hallmark of hypophosphatasia (HPP), but low readings of ALP are not always recognized in clinical routine. Understanding the clinical presentations associated with low ALP may contribute to a timelier diagnosis of HPP. METHODS: Data from paediatric patients with low ALP, excluding patients in intensive care and with oncological/haematological disorders, were analysed. Most recent ALP values, previous diagnoses, medication and relevant symptoms were extracted from patient records at nine specialised centres and analysed descriptively. A relationship between body height and ALP values was scrutinised by linear regression. RESULTS: Of 370 children, 15 (4.1%) had a diagnosis of HPP. In the subgroup without a diagnosis of HPP, 241 (67.9%) out of 355 patients had one or more medical conditions known to be associated with low serum ALP. Of those, hypothyroidism, malnutrition and steroid administration were most frequent. Characteristic symptoms, particularly, short stature, muscle weakness and delay of motor development were more frequent and ALP values were lower in patients with documented HPP diagnosis compared to patients without diagnosis of HPP (Ø z-scores: -2.52) (interquartile range [IQR] = 0.20) vs. -1.96 (IQR = 0.87). A weak positive linear relationship between z-scores of ALP and body height was identified (p<0.001). CONCLUSIONS: This analysis of paediatric patient records elucidates a wide range of disorders associated with low ALP activity. In case of additional specific symptoms, HPP should always be considered as a differential diagnosis.

Efficacy of low-dose 5-fluorouracil/salicylic acid in actinic keratoses in relation to treatment duration.

BACKGROUND: In Germany, the number of actinic keratoses (AKs) diagnosed and dermatologically treated has steadily risen in recent years. As AKs represent an early form of squamous cell carcinoma, prompt initiation of treatment is pivotal. The objective of this non-interventional study (NIS) was to determine the efficacy of a low-dose (0.5 %) fluorouracil/10 % salicylic acid (low-dose 5-FU/SA) solution in the treatment of AKs (clinical grade I and II according to Olsen) in relation to treatment duration. PATIENTS AND METHODS: This NIS followed 1,051 AK patients from 212 centers who were treated with low-dose 5-FU/SA. Data were collected at the start of treatment, at an optional follow-up appointment during the treatment period, at the end of treatment, and during the final assessment. RESULTS: During the observation period, there was a decrease in the mean number of lesions by approximately 70 % and in mean size by approximately 80 %. Roughly one-half of the patients were treated for less than six weeks. In the final global assessment, efficacy was rated as very good or good in approximately 89 % of patients; tolerability as very good or good in 87 %. CONCLUSIONS: Under routine clinical conditions, treatment with low-dose 5-FU/SA effectively reduced AK lesions even during short treatment periods, while exhibiting good tolerability. These findings are in keeping with results obtained from a clinical phase III trial.