Subcutaneous Immunoglobulin: A Newly Approved Treatment Option for CIDP

David Saperstein, MD
Co-Director GBS/CIDP Center of Excellence
Phoenix Neurological Associates
Phoenix, AZ

Intravenous immunoglobulin (IVIg) is one of the main therapies used to treat CIDP. This therapy can be very effective but there are drawbacks. Some people experience headaches related to the intravenous infusions. Some people have difficulty with IV access. Also, the need for IV infusions lasting several hours one or more days every month can prove burdensome with respect to work or travel. In recent years there has been growing interest in infusing immunoglobulin through the skin in a process called subcutaneous administration (subcutaneous immunoglobulin, or SCIg). SCIg is commonly used in patients with immunodeficiency. SCIg is administered by patients themselves at home. Infusions are generally given in the fat under the skin in the stomach or thighs. Over the last few years there have been small studies of SCIg in patients with CIDP. It has become a popular strategy for treating CIDP in Europe and has been used in some patients in the United States. Several brands of IVIg can be given subcutaneously, but there are a couple of products made specifically for subcutaneous administration. These are 20% in concentration, in contrast to most IVIg products which are 10%. The more concentrated 20% products allow infusions to be completed in just a couple of hours one or two days each week.  Unlike IVIg which is usually given in a big dose several weeks apart, SCIg must be administered every week).

The FDA recently approved a 20% formulation of SCIg called Hizentra for maintenance treatment of CIDP. This is based on the results of a large international study of Hizentra called the PATH study (for polyneuropathy and treatment with Hizentra). One hundred and seventy-two patients from North America, Europe, and Asia were studied. These were all patients who were receiving regular maintenance treatment with IVIg. To be eligible to participate in the PATH study, patients had to demonstrate that they needed regular treatment with IVIg. This required them to come off of IVIg and display objective worsening within 12 weeks. Of 245 patients taken off of IVIg, all but 28 worsened within 12 weeks. Ultimately, 172 patients were enrolled. 57 received low dose SCIg (0.2 grams per kilogram per week), 58 received high dose SCIg (0.4 grams per kilogram per week) and 57 received placebo (albumin protein solution). The patients were watched closely over the next 6 months. The main outcome was to see how many patients in each group worsened. Since each patient in the study had already shown that they worsened when coming off of regular IVIg treatments it was assumed that most of the patients in the placebo group would worsen. Interestingly, only 63% did. It is not clear why more did not. Nevertheless, many more patients in the placebo group relapsed over 6 months compared to patients who got SCIg (39% in the low dose group and 33% in the high dose group). Both dose groups did significantly better than placebo, but there was no significant difference between the low and high dose groups. Not surprisingly, the patients receiving SCIg had more skin irritation than patients getting placebo (19% in the low dose group, 29% in the high dose group and 7% in the placebo group). However, much fewer patients experienced reactions to Hizentra than they did to IVIg.

Approximately 90% of subjects rated the SCIg injection process as being easy to learn. About 53% of patients receiving SCIg preferred this treatment to IVIg, citing increased independence and less side effects.

Anybody with CIDP currently receiving IVIg could potentially be switched to SCIg. Of course, if the IVIg is working fine there is no reason to change anything. SCIg is a particularly appealing option for patients who get side-effects such and headaches or nausea from IVIg, who have trouble with IV access, or who prefer to have control of their treatment.  Another advantage of SCIg is that it can be stored at room temperature, so people can easily take it with them if they are travelling.

FDA approves CSL Behring treatment for CIDP

The Food and Drug Administration granted marketing clearance Friday for Hizentra, a treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) developed by CSL Behring.  Lisa Butler, executive director of the GBS/CIDP Foundation International, said “The approval of Hizentra offers patients who were once burdened by traveling to the infusion center or hospital the flexibility to self-administer their treatment at a time, place, and on a schedule that’s convenient for them.”

Read Full Article in Philadelphia Business Journal, March 16 2018.

Hansa Medical receives FDA Orphan Drug Designation for IdeS and the treatment of Guillain-Barré syndrome

February 16, 2018

Hansa Medical today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to IdeS (INN: Imlifidase) for the treatment of Guillain-Barré syndrome (GBS).

Hansa Medical, a Swedish company, is developing a drug, IdeS, which inactivates IgG. This inactivation would be like an instant plasmapheresis treatment. In February 2017, preclinical data from Hansa Medical showed that in an animal model of GBS, inactivation of IgG by IdeS treatment significantly promoted the recovery and reduced the degeneration of peripheral nerves. These data suggest that treatment with IdeS could potentially become a novel therapeutic strategy for the treatment of GBS. Several members of our GMAB are working with Hansa Medical to develop a study in GBS

Read Full Article

More information about the ODD has been posted on .

To All Those With Chronic Inflammatory Demyelinating Polyneuropathy

Recently, one of the physicians on the GBS/CIDP Foundation Medical Advisory Board saw a man who carried the diagnosis of CIDP for 2 years. During that time, he received IVIg monthly for 18 months then plasmapheresis monthly for five months without any clinical improvement. He went to a GBS/CIDP Foundation Center of Excellence where it was determined that he did not have CIDP, as the diagnosis was made on incorrectly interpreted nerve conduction studies in the wrong clinical context. The patient calculated that the cost of his treatments was over $400,000. Unfortunately, this is not uncommon.

Members of the Medical Advisory Board have been giving direct-to-patient talks on CIDP to Foundation patient support groups around the country. The main message is that the diagnosis and treatment of CIDP can and must be improved.

We want those with CIDP to:

  • Get the correct diagnosis,
  • Get the right treatment, and then
  • Get off treatment if treatment is no longer necessary.

It is important to know what CIDP is. Typical CIDP is a spectrum of polyneuropathies characterized by:

  • Development and progression for at least 2 months,
  • Sensory and motor symptoms,
  • Distal sensory loss, distal and proximal limb weakness, and areflexia on neurologic examination,
  • Demyelination on nerve conduction studies,
  • Elevated CSF protein without increase in cell count,
  • Normal routine blood studies including protein studies,
  • Nerve biopsy, if done, demonstrating T cell infiltration and macrophage-associated demyelination, and
  • Clear response to treatment(s) that have been demonstrated to be effective in CIDP.

There are 3 proven first line treatments for CIDP: IVIg, plasmapheresis and corticosteroids. The majority of people with true CIDP improve with one of these treatments. A clear response to any of these treatments is almost always seen within 2-3 months; no response after several months suggests rethinking the diagnosis and not continuing with that particular therapy.

Depending on the response to the first line treatments, other treatments may not be needed, but if other drugs are needed, a large number have been used successfully. Once on a successful treatment, it should be discontinued slowly to determine if it is still needed, because clinical trials have shown that 40% or more of those on treatments for CIDP do not require long-term therapy.

We recommend that you obtain another opinion for your CIDP from a Center of Excellence if:

  • You do not have the typical features of CIDP described above but are receiving treatment(s) for CIDP.
  • CIDP was diagnosed mainly based on nerve conduction studies.
  • CIDP was diagnosed mainly based on elevated spinal fluid protein.
  • You are not clearly improving on your current treatment(s).
  • You have been on treatments for a long time and are stable.

We hope these recommendations improve the care and treatment of those with CIDP. Questions can be directed to the Foundation.

David R. Cornblath, MD
Member, Medical Advisory Board
Member, Board of Directors

Kenneth C. Gorson, MD
Member, Medical Advisory Board