Lenabasum for Diffuse Cutaneous Systemic Sclerosis

Systemic Sclerosis (SS) is a rare disease caused by the immune system attacking the tissue which lines underneath the skin and internal organs (connective tissue). The exact cause of SS is not known but it is thought to happen when immune cells attack the body’s own tissues leading to the cells in the connective tissue producing too much collagen which causes scaring and thickening of the tissue. There are 2 types of SS; limited cutaneous SS (a milder form of SS affecting only parts of the body) and diffuse cutaneous SS (a more severe form of SS which can affect the whole body). The main symptoms of diffuse cutaneous SS are hardening of the skin, acid reflux, vomiting and diarrhoea, muscular pain, weakness and cramps. The outlook for people with diffuse cutaneous SS is generally poor due to high risk of life threatening complications such as heart, lung and kidney problems.


http://www.io.nihr.ac.uk/report/lenabasum-for-diffuse-cutaneous-systemic-sclerosis/


from the NIHR Innovation Centre

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency [HST7]

New:  Highly specialised technologies guidance


Strimvelis is recommended, within its marketing authorisation, as an option for treating adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID) when no suitable human leukocyte antigen-matched related stem cell donor is available.

Pirfenidone for treating idiopathic pulmonary fibrosis [TA 504]

New: Technology appraisal guidance




https://www.nice.org.uk/guidance/ta504Pirfenidone is recommended as an option for treating idiopathic pulmonary fibrosis in adults only if:

• the person has a forced vital capacity (FVC) between 50% and 80% predicted
• the company provides pirfenidone with the discount agreed in the patient access scheme and
• treatment is stopped if there is evidence of disease progression (an absolute decline of 10% or more in predicted FVC within any 12‑month period).
• 
  

Pirfenidone (Esbriet, Roche) is an oral immunosuppressant with anti-inflammatory and antifibrotic effects.

NIHR Signal Biological therapies for psoriasis do not increase serious infection risk

People with psoriasis who take an immune-modulating treatment are no more likely to get serious infections than people taking standard therapies.inf




There are fears that these biological therapies raise the risk of serious infections and this has discouraged their use. They are recommended by NICE for moderate to severe psoriasis. Previous studies have reached conflicting conclusions, making it hard to advise on the true risk.




This study used a large database of people with psoriasis from the UK and Ireland. It compared serious infection risk of the biological therapies (etanercept, adalimumab or ustekinumab) with non-biological therapies, after accounting for factors such as other illnesses. It found none of the biological therapies studied had a higher risk of infection compared to non-biological therapies or compared to each other.




From the NIHR Dissemination Centre

Risankizumab (by Subcutaneous Injection) for Moderate to Severe Chronic Plaque Psoriasis

Risankizumab is a drug which is injected into the skin. It works in a unique way by blocking a specific process which allows the body’s immune cells (specifically T-cells) from attacking and damaging the skin. Risankizumab is currently being trialled in a range of diseases involving the immune system including Crohn’s disease, and psoriatic arthritis.

From the NIHR Innovation Observatory

Ponesimod for Relapsing-remitting Multiple Sclerosis

Ponesimod is a drug which works by blocking the signals which allow the body’s immune cells to travel to and damage the nerve cells. By preventing the immune cells from damaging the nerves, it is thought this drug will stop the damage which causes MS ‘relapses’. In clinical trials it has been shown that ponesimod reduces the number of ‘relapses’ in people with MS and reduced the amount of damage to the nerves (measured by brain scans) compared to a placebo.

From the NIHR Innovation Observatory

Screening committee recommends trial of testing babies for SCID

Following a review of the evidence, the independent expert screening committee recommends that screening for severe combined immunodeficiency (SCID) should be tried for a period of time in the NHS.

SCID refers to a number of rare inherited conditions which affect the development of a baby’s white blood cells – these are an important part of the immune system and make it difficult for babies to fight infections. Around 15 to 25 babies are born with the condition every year in the UK. The treatment is a bone marrow transplant, which can repair the damaged immune system.

The trial period will allow the committee to gather information about the practicalities and likely effect of screening before a final recommendation is made on whether to include SCIDin the NHS newborn bloodspot screening programme.

PO199 An autoimmune cranial and peripheral polyneuropathy with myositis

Association of British Neurologists (ABN) Annual Meeting 20172,3–5th May 2017 ACC, Liverpool

A 69 year old man presented in 2006 with diplopia, facial weakness, bulbar dysfunction, profound sensory motor neuropathy (distally more than proximally), and hyporeflexia. Blood at the time did not reveal any major abnormalities. Electrophysiology confirmed severe sensory and motor axonal polyneuropathies. Sural nerve biopsy was non-specific, whereas muscle biopsy specifically showed peri-vascular inflammatory cells. Following significant improvement with intra-venous methylprednisolone, he returned to a functional independent life. More debilitating similar episodes recurred six and nine years later with involvement of cranial nerves 2–7, 9–12, accompanied by predominantly upper limb weakness and hyporeflexia. Post-contrast neural MR and CSF constituents remained normal (negative antiganglioside antibodies). Electrophysiology confirmed progressive axonal peripheral polyneuropathy. Liver biopsy ruled out cirrhosis/autoimmune hepatitis. In 2016, repeat Ro antibodies were positive, with3-year history of Raynaud’s phenomenon. Patient was treated with immunoglobulin, intravenous Methylprednisolone, followed by oral steroids. Subsequently, intravenous Cyclophosphamide then Mycophenolate with ongoing tapered steroids resulted in good response, despite persistent muscle wasting without fasciculations.

UHCW Research: Akram A Hosseini, Fizzah Ali, Shirish Dubey, and Antony Thomas

PO108 Susac syndrome: a case for early, aggressive and sustained treatment

Association of British Neurologists (ABN) Annual Meeting 20172,3–5th May 2017 ACC, Liverpool

We report a case of Susac syndrome, initially suspected to be multiple sclerosis, and advocate diagnostic caution and high suspicion. We describe a successful therapeutic approach comprising corticosteroids, intra-venous immunoglobulin and cyclophosphamide for encephalopathic relapse of Susac syndrome.

UHCW Research: Fizzah Ali, Akram A Hosseini and Antony Thomas

Ibrutinib for treating Waldenstrom’s macroglobulinaemia [TA491]

New technology appraisal guidance from NICE:

Evidence-based recommendations on ibrutinib (Imbruvica) for treating Waldenstrom’s macroglobulinaemia in adults after at least 1 previous therapy.

Ibrutinib is recommended for use in the Cancer Drugs Fund as an option for treating Waldenstrom's macroglobulinaemia in adults who have had at least 1 prior therapy, only if the conditions in the managed access agreement for ibrutinib are followed.

Pembrolizumab (KEYTRUDA®) for advanced, metastatic oesophageal cancer – second line

The most common treatment options for oesophageal cancer in the UK is surgery; alternative treatments are radiotherapy and chemotherapy. Pembrolizumab is a type of immunotherapy, which works targeting specific proteins that stimulates an immune response that targets the cancer cells. It increases the body’s natural ability to identify and attack cancer cells. If licenced, pembrolizumab will offer an additional treatment option for patients with oesophageal cancer and prolong the time without cancer progression.


From the NIHR Innovation Observatory

Nivolumab (Opdivo) with ipilimumab (Yervoy) for recurrent, metastatic, squamous cell head and neck cancer – first line

Nivolumab is a type of protein designed to attach to a certain type of white blood cells called the T cells. T cells are part of the immune system needed to attack the cancer. Nivolumab acts to improve the activity of T cells, thereby increasing the ability of the immune system to kill cancer cells. Ipilimumab is another type of protein that acts in a different way to increase the activity of T cells. If licenced, nivolumab in combination with ipilimumab will offer additional treatment option to prolong lives of this patient group.

Immunosuppressive therapy for kidney transplant in children and young people [TA482]

New:  Technology Appraisal Guidance

Evidence-based recommendations  on immunosuppressive therapies for preventing kidney rejection in children and young people. The therapies are basiliximab (Simulect), immediate-release tacrolimus (Adoport, Capexion, Modigraf, Prograf, Tacni, Vivadex), mycophenolate mofetil (Cellcept and non-branded versions), rabbit anti-human thymocyte immunoglobulin (Thymoglobuline), prolonged-release tacrolimus (Advagraf, Envarsus), mycophenolate sodium (Myfortic, Ceptava), sirolimus (Rapamune), everolimus (Certican) and belatacept (Nulojix).

This guidance makes recommendations on using basiliximab, rabbit anti-human thymocyte immunoglobulin, tacrolimus (immediate-release and prolonged-release), mycophenolate mofetil, mycophenolate sodium, sirolimus, everolimus and belatacept after kidney transplant in children and young people. The recommendations apply only to the initial immunosuppressive therapy (induction and maintenance therapy) started around the time of kidney transplant.

It was outside the scope of the appraisal to make recommendations on using azathioprine or corticosteroids after kidney transplant in children and young people.
Under an exceptional directive from the Department of Health, the appraisal committee was allowed to make recommendations about using drugs outside the terms of their marketing authorisations if there was compelling evidence of their safety and effectiveness.

NICE approves gene therapy for rare ‘bubble baby syndrome’

Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’ has been approved by NICE in draft guidance.

Immunosuppressive therapy for kidney transplant in children and young people [TA481]

New Guidance from NICE:

Evidence-based recommendations on immunosuppressive therapies for preventing kidney rejection in children and young people. The therapies are basiliximab (Simulect), immediate-release tacrolimus (Adoport, Capexion, Modigraf, Prograf, Tacni, Vivadex), mycophenolate mofetil (Cellcept and non-branded versions), rabbit anti-human thymocyte immunoglobulin (Thymoglobuline), prolonged-release tacrolimus (Advagraf, Envarsus), mycophenolate sodium (Myfortic, Ceptava), sirolimus (Rapamune), everolimus (Certican) and belatacept (Nulojix).

This guidance makes recommendations on using basiliximab, rabbit anti-human thymocyte immunoglobulin, tacrolimus (immediate-release and prolonged-release), mycophenolate mofetil, mycophenolate sodium, sirolimus, everolimus and belatacept after kidney transplant in children and young people. The recommendations apply only to the initial immunosuppressive therapy (induction and maintenance therapy) started around the time of kidney transplant.
It was outside the scope of the appraisal to make recommendations on using azathioprine or corticosteroids after kidney transplant in children and young people.
Under an exceptional directive from the Department of Health, the appraisal committee was allowed to make recommendations about using drugs outside the terms of their marketing authorisations if there was compelling evidence of their safety and effectiveness.

Immunosuppressive therapy for kidney transplant in adults

New Guidance from NICE:

Evidence-based recommendations on immunosuppressive therapies for preventing kidney rejection in children and young people. The therapies are basiliximab (Simulect), immediate-release tacrolimus (Adoport, Capexion, Modigraf, Prograf, Tacni, Vivadex), mycophenolate mofetil (Cellcept and non-branded versions), rabbit anti-human thymocyte immunoglobulin (Thymoglobuline), prolonged-release tacrolimus (Advagraf, Envarsus), mycophenolate sodium (Myfortic, Ceptava), sirolimus (Rapamune), everolimus (Certican) and belatacept (Nulojix).

This guidance makes recommendations on using basiliximab, rabbit anti-human thymocyte immunoglobulin, tacrolimus (immediate-release and prolonged-release), mycophenolate mofetil, mycophenolate sodium, sirolimus, everolimus and belatacept after kidney transplant in children and young people. The recommendations apply only to the initial immunosuppressive therapy (induction and maintenance therapy) started around the time of kidney transplant.
It was outside the scope of the appraisal to make recommendations on using azathioprine or corticosteroids after kidney transplant in children and young people.
Under an exceptional directive from the Department of Health, the appraisal committee was allowed to make recommendations about using drugs outside the terms of their marketing authorisations if there was compelling evidence of their safety and effectiveness.

‘Vaginal seeding’ birth trend could do more harm than good, say experts

The potential benefits of vaginal seeding do not outweigh the risks, according to a group of doctors who reviewed the available evidence published in BJOG: An International Journal of Obstetrics and Gynaecology.

Vaginal seeding is the practice of exposing babies born by caesarean section to their mother’s vaginal fluids in order to expose them to bacteria that may help to build their immunity against some chronic conditions, such as asthma and allergies. This involves taking a swab of vaginal fluid and applying it to the baby’s eyes, face and skin after birth.

Screening consultation on rare but serious condition in babies

Consultation launched on whether screening for Severe Combined Immune Deficiency (SCID) in babies should be tried within the NHS.

Skin involvement in systemic sclerosis: rituximab

This evidence summary from NICE includes 7 studies that investigated rituximab (usually 375 mg/m2 weekly for 4 weeks at 0 and 6 months, or 1,000 mg at 0 and 2 weeks) for treating skin involvement in systemic sclerosis (mainly diffuse).

Perivillous fibrin deposition recurrence

Answer to the question Is there any treatment recommended to reduce the risk of recurrent perivillous fibrin deposition/maternal floor infarction in women with no evidence of thrombophilia/ antiphospholipid syndrome?, posted to the RCOG query bank.