Hurler syndrome surgery

Revision as of 13:45, 19 July 2016 by Irfan Dotani (talk | contribs)
(diff) ← Older revision | Latest revision (diff) | Newer revision → (diff)
Jump to navigation Jump to search

Hurler Syndrome Microchapters

Home

Patient Information

Overview

Historical Perspective

Classification

Pathophysiology

Causes

Differentiating Hurler Syndrome from other Diseases

Epidemiology and Demographics

Risk Factors

Screening

Natural History, Complications and Prognosis

Diagnosis

History and Symptoms

Physical Examination

Laboratory Findings

Electrocardiogram

X Ray

CT

MRI

Echocardiography or Ultrasound

Other Imaging Findings

Other Diagnostic Studies

Treatment

Medical Therapy

Surgery

Primary Prevention

Secondary Prevention

Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

Hurler syndrome surgery On the Web

Most recent articles

Most cited articles

Review articles

CME Programs

Powerpoint slides

Images

American Roentgen Ray Society Images of Hurler syndrome surgery

All Images
X-rays
Echo & Ultrasound
CT Images
MRI

Ongoing Trials at Clinical Trials.gov

US National Guidelines Clearinghouse

NICE Guidance

FDA on Hurler syndrome surgery

CDC on Hurler syndrome surgery

Hurler syndrome surgery in the news

Blogs on Hurler syndrome surgery

Directions to Hospitals Treating Hurler syndrome

Risk calculators and risk factors for Hurler syndrome surgery

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Govindavarjhulla, M.B.B.S. [2]

Please help WikiDoc by adding content here. It's easy! Click here to learn about editing.

Overview

Bone marrow transplantation (BMT) and umbilical cord blood transplantation (UCBT) can successfully treat the mucopolysaccharidoses. Abnormal physical characteristics, except for those affecting the skeleton and eyes, can be improved, and neurologic degeneration can often be halted. BMT and UCBT are high-risk procedures with high rates of morbidity and mortality. However, they are the only treatments that have the potential to cure the disease.

Surgery

Bone Marrow Transplantation

  • These procedures lead to replacement of mutated/ non-functional cells of the bone marrow with functional cells.
  • Functional new cells act as a source for the deficient enzyme.
  • Transplant has the best outcome when its done as soon as the diagnosis is established.
  • Children who receive a transplant early enough can have normal or near-normal mental development.
  • If the disease has progressed causing organ damage, the child is at high risk of developing life-threatening complications from transplant.
  • Transplants cannot undo the damage done to nervous system.
  • Risks of transplant

Umbilical Cord Blood Transplantation

  • This plays a crucial role when suitable donor is not available for transplant.
  • In a study done between 1995 and 2002, quite a convincing results were obtained with the cord blood transplants.[1]
  • Cord blood transplantation favorably altered the course of disease.[1]

References

  1. 1.0 1.1 Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J (2004). "Cord-blood transplants from unrelated donors in patients with Hurler's syndrome". The New England Journal of Medicine. 350 (19): 1960–9. doi:10.1056/NEJMoa032613. PMID 15128896. Retrieved 2013-02-25. Unknown parameter |month= ignored (help)

Template:WS Template:WH