Paediatric advice on clinical support and homecare (updated 26 March)

Updated: Mar 26, 2020

The clinical centres are working tirelessly to manage the huge volumes of calls and enquiries from our patient communities. For many they have had to either cancel all clinics and non-emergency appointments or move them to telephone appointments. These are unprecedented times and many of our doctors and nurses have already been, or are on standby, to be called to the front line. 

Each centre has an answerphone to ensure that they do not miss any calls. Please bear with them as they filter through the many calls that are coming through on a daily basis. 

The current advice is that critically ill LSD patients should not be transferred to the specialist centres and should continue to receive treatment at their local hospital as appropriate. Advice and support should be sought by your specialist centre as required. Keep to hand:

  • your specialist centre telephone numbers

  • any medical alert cards

  • care plans or leaflets for medical staff that explain your condition.

Speak with your closest family members and advocates to ensure they know where information is kept.

Please be aware that advice is changing daily and it is important that you keep yourselves up to date with the latest government advice.

Extremely vulnerable high-risk patients

Clinical centres are already reviewing all their patients and will be in contact with those patients deemed to be high risk to discuss individual circumstances. Please read the government advice on shielding and protecting people defined on medical grounds as extremely vulnerable from COVID-19.

Self-isolation and social distancing

Social distancing - vulnerable groups, families and everyone

Social distancing measures are steps you can take to reduce the social interaction between people. The government is currently asking all people to social distance themselves. This is to help reduce the transmission of coronavirus (COVID-19). Please see the government website for up to date information.

Self-isolation - symptomatic and unwell or someone you live with is symptomatic

In this instance you should self-isolate at home. Please see the goverment and NHS self-isolation advice for up to date information on what this means.

When to trigger self-isolation/shielding

The paediatric centres will follow government advice and support patients who are shielding or choose to self-isolate based on government advice. Specialist centres may contact some families to clarify whether their underlying condition constitutes a high risk group that would benefit from shielding but this will be highly individual, patient-specific advice.

Homecare/enzyme replacement therapy (ERT) infusions

If a patient is self-isolating because of COVID-19 symptoms

Nurse visits for ERT infusions are paused for the duration of self-isolation (currently 14 days). If patients can become independent that would be advantageous, but homecare resources are not available for large-scale, short-term training.

If a family chooses to strictly self-isolate because of underlying vulnerability

Your specialist centre will have an individual discussion with you about ERT and the risk/benefit balance of taking a prolonged "drug holiday". They will offer reassurance to patients and families undergoing a "drug holiday" that this is a decision in their best interest, based on the greater risk to them of being infected with COVID-19, than any modest or moderate effect of missing treatment for a period of time.

If a family chooses to undertake "more stringent social distancing"

Because of underlying vulnerability, but not full family self-isolation, it may be appropriate for some patients to continue receiving ERT as long as homecare companies can deliver this. This is felt to be essential for:

  • Infantile LAL Deficiency

  • Infantile Pompe Disease

  • Type III Gaucher

  • some MPS patients being prepared for stem cell transplantation.

CLN2 patients receiving intrathecal cerliponase in hospital will also continue to be, prioritised for this treatment.

If Homecare Company staffing becomes critically low, then patients with other diseases may be required to reduce their dosing frequency.

Patients with indwelling venous access devices who are having a drug holiday