Primary immunodeficiency diseases (PIDD) comprise a group of 200+ immune disorders wherein a part of the body’s immune system is either absent or does not function properly. The conditions, which result because of a single-gene defect, are passed down from either one or both parents. In the most common form, PIDDs occur due to problems in DNA that result in a lack of white blood cells, which the immune system is composed of.

Regardless of their difference in expression, PIDDs are characterized by an increased susceptibility to infection. If left untreated or if not diagnosed properly, PIDDs can have repercussions such as chronic illness, disability, reduction in working capacity, diminished quality of life for patients and their families, permanent damage to organs, and even death. Nevertheless, PIDDs are rare and are prevalent in 1:10,000 live births, not including IgA (immunoglobulin A) deficiency prevalence statistics.

Transparency Market Research expects the global immunodeficiency diseases market to expand at a CAGR of 6.1% for the 2015-2023 period. This will result in the market being valued at US$7.6 bn by 2023, increasing from US$4.4 bn in 2014.

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Stem Cell Treatment and Gene Therapy to Lead PIDD Market

Immunoglobulin replacement therapy, stem cells, antibiotic therapy, gene therapy, and others are the major treatment lines employed for PIDDs. The utilization pattern of these treatment lines is charted by TMR analysts as follows:

1. Immunoglobulin (Ig): This therapy, employed for over two decades, is used as a course of treatment for hypogammaglobulinaemia. Lately, Ig has also been used in combination with other treatments for the treatment of immunodeficiency and partial antibody deficiency. Ig is a replacement therapy and is predominantly administered through either intravenous or subcutaneous modes. For example, as per a survey conducted by the Medical Insights Group in 2014, in the U.S., 54% of home-based Ig is delivered through subcutaneous (SC) mode and the remaining 46% through IV. SC mode is exhibiting elevated demand, especially among young individuals. It can be delivered in home settings and needs just one hour of infusion time, a significant advantage over IV, which requires three to four hours of infusion time in a clinical environment.

2. Antibiotic Therapy: Long-term prophylactic antibiotics are widely used as either a primary or supplementary therapy for PIDD. This line of treatment has particularly transformed clinical outcomes for chronic granulomatous disease, Wiskott-Aldrich syndrome, complement deficiencies, and hyper IgE syndrome. Penicillin, macrolides, sulfonamides, aminoglycosides, cephalosporins, fluoroquinolones, and tetracyclines are commonly used antibiotics for PIDD.

3. Hematopoietic Stem Cell Treatment (HSCT): This successfully cures a host of immunodeficiency disorders; with advancement in medical science, HSCT offers an improved survival rate and better life quality, particularly for orphan diseases.

4. Gene Therapy: This is used for individuals with severe combined immunodeficiency (SCID) due to X-linked SCID, Wiskott-Aldrich syndrome, adenosine deaminase deficiency, and chronic granulomatous disease.

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Gene therapy and stem cell treatment will display the fastest growth in the primary immunodeficiency diseases market in the coming years due to the increasing number of research undertakings for improved outcomes and a significant drop in morbidity and mortality rates in the past few years.

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