Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
| Status: | Terminated |
|---|---|
| Conditions: | Other Indications, Neurology, Neurology, Endocrine, Hematology, Metabolic |
| Therapuetic Areas: | Endocrinology, Hematology, Neurology, Pharmacology / Toxicology, Other |
| Healthy: | No |
| Age Range: | Any - 21 |
| Updated: | 12/30/2017 |
| Start Date: | March 2008 |
| End Date: | February 2010 |
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation
The primary objective of this clinical trial is to evaluate the ability to achieve and
sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of
metabolism undergoing hematopoietic stem cell transplantation (HCT).
sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of
metabolism undergoing hematopoietic stem cell transplantation (HCT).
This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is
a new protocol to take the place of several older protocols. While survival has been very
good on the prior protocols over the past decade, incomplete engraftment has remained
somewhat problematic. Therefore, we have modified the preparative regimen somewhat to
increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that
is more immune suppressive. In addition, we have modified the supportive care regimen. Based
on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers
of inflammation and oxidative stress for the families that consent to these research studies.
a new protocol to take the place of several older protocols. While survival has been very
good on the prior protocols over the past decade, incomplete engraftment has remained
somewhat problematic. Therefore, we have modified the preparative regimen somewhat to
increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that
is more immune suppressive. In addition, we have modified the supportive care regimen. Based
on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers
of inflammation and oxidative stress for the families that consent to these research studies.
Inclusion Criteria:
- Mucopolysaccharidosis (MPS) Disorders:
- MPS IH (Hurler syndrome)
- MPS-VI (Maroteaux-Lamy syndrome)
- MPS VII (Sly syndrome).
- Glycoprotein metabolic disorders:
- Alpha mannosidosis
- Fucosidosis
- Aspartylglucosaminuria
- Sphingolipidoses and Recessive Leukodystrophies: Presymptomatic patients with globoid
cell leukodystrophy (GLD, also known as Krabbe disease) and metachromatic
leukodystrophy (MLD) will be eligible for treatment on this protocol. White matter
disease by magnetic resonance imaging (MRI) alone is not an exclusion if the patient
is asymptomatic.
- Peroxisomal Disorders: Presymptomatic patients with inherited peroxisomal disorders
associated with of very long chain fatty acids (VLCFA) elevation, identified by family
history or laboratory testing (including neonatal screening), are eligible for this
protocol. White matter disease by MRI alone is not an exclusion if the patient is
asymptomatic.
- Other Inherited Diseases of Metabolism:
- Wolman syndrome (acid lipase deficiency)
- Niemann-Pick B patients (sphingomyelin deficiency)
- Niemann-Pick C subtype 2
- Donor Availability: Patients considered for transplantation must have a sufficient
graft as based on current criteria of the University of Minnesota Blood and Marrow
Transplantation Program: Priority will be as follows, although in circumstances in
which timing is of the essence, cord blood grafts may be chosen over an unrelated
graft, despite the priority listed above.
- Multidisciplinary Evaluation: Patients will be eligible for transplantation only after
they are seen and evaluated by members of the Inherited Metabolic and Storage Disease
Program (IMSD) team, and the team has offered transplantation to the patient/family.
Exclusion Criteria:
- Symptomatic patients with peroxisomal or lysosomal disorders are excluded but may be
considered for other treatment protocols.
- Major organ dysfunction. Evidence of major organ impairment, including:
- Cardiac: left ventricular ejection fraction <40%
- Renal: serum creatinine >2.5 x normal for age
- Hepatic: total bilirubin >3 x normal, or Alanine transaminase (ALT) > 3 x normal
- Pulmonary: requirement for continuous oxygen supplementation
- Pregnancy
- Evidence of human immunodeficiency virus (HIV) infection or known HIV positive
serology
- Patients >21 years of age.
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