MPS Surgery: A Hope for Children With Rare Diseases

What is MPS Surgery?

MPS surgery, also known as hematopoietic stem cell transplantation (HSCT), is a medical procedure that involves transplanting healthy stem cells into a patient with a rare genetic disorder called mucopolysaccharidosis (MPS).

MPS is a group of inherited conditions that affect the body's ability to break down certain complex sugars called glycosaminoglycans (GAGs). This can lead to a buildup of GAGs in various tissues and organs, resulting in a wide range of symptoms, including skeletal deformities, developmental delays, intellectual disabilities, and organ damage.

HSCT is a potentially curative treatment for MPS. It involves harvesting stem cells from a healthy donor, such as a sibling or an unrelated donor from a national registry. These stem cells are then infused into the patient's bloodstream, where they travel to the bone marrow and begin to produce healthy blood cells.

Benefits of MPS Surgery

HSCT can provide significant benefits for children with MPS. These benefits include:

• Improved physical function, including increased mobility and reduced pain
• Improved cognitive function, including increased IQ and attention span
• Reduced risk of organ damage and other complications
• Extended life expectancy

Risks of MPS Surgery

As with any medical procedure, HSCT carries some risks. These risks include:

• Graft-versus-host disease (GVHD), a condition in which the donor's immune cells attack the patient's tissues
• Infections
• Bleeding
• Death

Who is a Candidate for MPS Surgery?

HSCT is not a suitable treatment for all children with MPS. The best candidates for HSCT are children who are diagnosed early and who have a compatible donor. Children who have severe organ damage or other complications may not be good candidates for HSCT.

How to Prepare for MPS Surgery

Preparing for HSCT involves a number of steps, including:

• Finding a compatible donor
• Undergoing a series of medical tests
• Receiving chemotherapy and radiation therapy to prepare the body for the stem cell transplant

What to Expect During MPS Surgery

HSCT is a complex procedure that typically takes place in a hospital setting. The procedure involves the following steps:

• Harvesting stem cells from the donor
• Preparing the patient for the transplant by giving them chemotherapy and radiation therapy
• Infusing the donor stem cells into the patient's bloodstream

Recovery from MPS Surgery

Recovery from HSCT can take several months. During this time, the patient will need to be closely monitored for complications. The patient may also need to take medications to prevent GVHD and other complications.

Conclusion

HSCT is a potentially curative treatment for children with MPS. It can provide significant benefits, including improved physical and cognitive function, reduced risk of organ damage, and extended life expectancy. However, HSCT is a complex procedure that carries some risks. It is important to carefully consider the risks and benefits before making a decision about whether to proceed with HSCT.