We read with curiosity a consensus statement on spinal muscular atrophy type 1 (SMA1) standard of care in the UK.(1) The authors stated that "non-invasive ventilation (NIV) is ineffective and contraindicated in infants who have severe bulbar weakness, usually present in babies with severe type 1 SMA," that is, in those with "onset of symptoms before 6 months of age". Ironically, the authors interpreted studies of prolonged survival using NIV for continuous ventilatory support for severe SMA 1 patients, floppy at birth, unable to receive any nutrition by mouth, averbal, and permanently and continuously ventilator dependent before 6 months of age, as not severely affected. In reality SMA1 infants who are continuously NIV dependent before 1 year of age always have their "onset" before 6 months of age. Indeed, most if not all are floppy at birth and 73 of 75 in one study (2) had paradoxical breathing even though the pediatricians do not always recognize it at birth. Even children with SMA type 2 who develop the ability to sit independently can have "onset of weakness" before 6 months of age. Thus, the timing of the disease "onset" is not valid for classifying disease severity since it depends on the experience and astuteness of individual mothers and pediatricians. Maximum ever achieved (plateau) vital capacity (VC), cry VC or otherwise, is a more important indicator for severity of disease, especially since, in the UK where NIV is "contraindicated in infants with severe bulbar weakness,"these patients invariably die of respiratory failure. To determine the plateau VC, however, necessitates that the children live long enough to plateau and are often, therefore, continuously NIV dependent. In a study of 103 severe to typical SMA type 1 patients, all of whom floppy at birth with essentially no bulbar-innervated muscle function and all having respiratory failure and requiring gastrostomy tubes before the second birthday including most before 8 months of age, many are now over age 15 despite requiring continuous NIV support for over 15 years. The plateau VCs were less than 100 ml in some cases and their current VCs less than 20 ml (<0.2% of normal). Despite this, they are alive and most without tubes in their necks.(2) While this paper (2) might have been in press while the consensus was being prepared, earlier papers on identically severe, typical SMA 1 children were available to the authors.(3,4) What would have been the fate of these "mild cases" according to the UK expert consensus?

References 1. Roper H, Quinlivan R. Implementation of "The Consensus Statement for the Standard of Care in Spinal Muscular Atrophy" when applied to infants with severe type 1 SMA in the UK. Arch Dis Child. 2009 2. Bach JR, Gupta K, Reyna M, Hon A. Spinal muscular atrophy type 1: prolongation of survival by noninvasive respiratory aids. doi:10.1089/pai.2009.0002; Pediatric Asthma, Allergy & Immunology. 2009,22(4):151-162. 3. Bach JR, Baird JS, Plosky D, Nevado J, Weaver B. Spinal muscular atrophy type 1: management and outcomes. Pediatr Pulmonol 2002;34:16-22. 4. Bach JR, Saltstein K, Sinquee D, Weaver B, Komaroff E. Long term survival in Werdnig-Hoffmann Disease. Am J Phys Med Rehabil 2007;86(5):339 -345 and Quiz 346-348, 379.

Conflict of Interest:

None declared

To the Editor,

we appreciated the meta-analysis by Fisher et al about specific oral tolerance induction (SOTI) in food allergic children (1), but we take exception to their statement about SOTI goal. The authors highlight as a substantial limit the fact that although SOTI increased the threshold of reactivity only few children were able to consume an age appropriate portion of the relevant food and fewer attempted to liberalize the dose up to extremely large quantities as happens in real life. We believe that this conclusion is inappropriate both from a methodological and a substantial point of view. As the former is concerned the length of follow up of the studies is too short and data are still too limited to allow such a statement . While being well accepted the fact that tolerance can no longer be considered an all or nothing phenomenon it is no common knowledge the fact that in the years in course of SOTI there is a constant and steady decrease of specific IgE for milk ,when assumption is continued, up to normal values. Even if not reported in long time by controlled studies (very difficult to design) this is a clear evidence from routine practice of our and other centres (2) practicing SOTI from years. This fact usually allows a progressive increase in milk assumption up to an unrestricted diet in many patients. The second exception is about the substantial issue of which is SOTI goal for patients and their families in real life. For people with severe allergy who has being living for years with the psychological burden of a life threatening event the assumption of even limited amounts of milk represents a paramount achievement in the perceived quality of life. Even after repeated reactions, with the impossibility of increase in the dose, families are highly motivated to remain to a fix safe dose . Furthermore many of these children have a kind of food aversion for milk and are not eager ( when not frightened) at all to assume age appropriate , extremely large amounts of milk derivates , at least in the first period of SOTI. Their main goal is to assume a protective dose to avoid risk from contaminations and allowing a normal life ( going to parties). We agree with Fisher that more trials are needed but we think that a pragmatical approach which takes into account families' needs must be maintained when considering SOTI goals.

1) HR Fisher, G du Toit, G. Lack. Specific oral tolerance induction in food allergic children: is oral desensitization more effective than antigen avoidance ? : A meta-analysis of published RCTs. Arch Dis Child online june 3, 2010.

2) Patriarca G., Nucera E, Pollastrini E, Roncallo C, De Pasquale T, Lombardo C et al. Oral Specific desensitization in food-allergic children. Dig Dis Sci. 2007;52:1662-72

Conflict of Interest:

None declared

Our long experience in the use of electronic reporting supports and extends the findings reported by Lynn et al (1). The Australian Paediatric Surveillance Unit (APSU) has used electronic reporting since 1997 to collect important information on up to 16 rare childhood diseases simultaneously, including rare genetic, metabolic, traumatic, communicable, vaccine-preventable and mental conditions (2). The paediatricians and paediatric sub-specialists who contribute to the APSU currently comprise 92% of Fellows in Paediatrics listed with the Royal Australasian College of Physicians (3).

Since the introduction of electronic reporting, the proportion of clinicians who receive and respond to monthly APSU report cards via e-mail has increased steadily from 11% of 918 clinicians in 1997 (2) to 82% of 1329 clinicians in 2010. The overall report card response rate has remained at or above 90% annually since 1994 (2).

In addition, in the recent evaluation of the APSU which included a survey of clinicians who report to the APSU, 67% of 818 respondents also reported willingness to provide detailed demographic and clinical data via a secure website if one was available (4). The APSU is currently developing such a web-based system. Importantly, electronic notification and data collection allows rapid response to outbreaks or importation of diseases by quickly issuing alerts on new or emerging conditions. In response to reports of influenza-related child deaths in 2007, APSU was able to rapidly initiate weekly surveillance for severe complications of seasonal influenza (5). The implementation of this system proved invaluable for the rapid collection of detailed data on severe complications of influenza during the H1N1 2009 pandemic.

Our experiences and the recent experience of Lynn and colleagues demonstrate that clinicians are willing participants in active disease surveillance which uses electronic reporting.

References

1. Lynn RM, Riding K, McIntosh N. The use of electronic reporting to aid surveillance of ADRs in children: a proof of concept study. Arch Dis Child 2010;95:262-265.

2. Srikanthan S, Zurynski Y, Elliott E. Australian Paediatric Surveillance Unit: Celebrating 15 years of surveillance. 1993-2007. APSU 2008.

3. He S, Zurynski YA, Elliott EJ. Evaluation of a national resource to identify and study rare diseases: The Australian Paediatric Surveillance Unit. J Paediatr Child Health 2009;45:498-504.

4. He S, Zurynski YA, Elliott EJ. What do paediatricians think of the Australian Paediatric Surveillance Unit? J Paediatr Child Health 2010; doi: 10.1111/j.1440-1754.2010.01755

5. Zurynski YA, Lester-Smith D, Festa MS, Kesson AM, Booy R, Elliott EJ. Enhanced surveillance for serious complications of influenza in children: role of the Australian Paediatric Surveillance Unit. Commun Dis Intell 2008;32:71-76.

Conflict of Interest:

None declared

I read with interest the article published by Archer et al on Multisource Feedback of paediatric registrars. This article is very encouraging for trainees like me and also answers doubts about the validity and reliability of such work place based assessments.

One of the main conclusions of the authors is that self selection of assessors by trainees should end. But this raises questions about the validity of an assessment which as reported by the authors is based on the length of working relationship, and trainees would be the best judge of choosing assessors with whom they have had a reasonable length of working relationship in terms of number of shifts worked. But as the authors report that unregulated self selection of assessors can lead to selection bias. A possible solution could be that the trainee provides a list of assessors and their supervisor then invites certain number of assessors selected randomly to provide feedback.

Another point to be considered in the same context of length of working relationship is the timing of the MSF sessions. If trainees are changing posts every six months which is still possible in the current training pattern it might be useful to have these sessions towards the later part of the post rather than in the middle as they currently are done.

One of the other questions raised by the authors is the seniority of the assessors which I feel is partly corrected by the current system of MSF which suggests that trainees should choose a certain number of consultants including their supervisor and certain number of nursing colleagues as well.

Conflict of Interest:

None declared