Radiologic resolution of malignant infantile osteopetrosis skeletal changes following hematopoietic stem cell transplantation

@article{HashemiTaheri2015RadiologicRO,
  title={Radiologic resolution of malignant infantile osteopetrosis skeletal changes following hematopoietic stem cell transplantation},
  author={Amir Pejman Hashemi Taheri and Amir Reza Radmard and Soheil Kooraki and Maryam Behfar and Neda Pak and Amir Ali Hamidieh and Ardeshir Ghavamzadeh},
  journal={Pediatric Blood \& Cancer},
  year={2015},
  volume={62}
}
Hematopoietic stem cell transplantation (HSCT) is the only known curative treatment of malignant infantile osteopetrosis (MIOP). In this study, short‐term serial bone surveys were used to assess radiologic evolution of skeletal changes after HSCT in MIOP. 

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References

SHOWING 1-10 OF 22 REFERENCES

Imaging of malignant infantile osteopetrosis before and after bone marrow transplantation

TLDR
Investigation of the radiological findings at presentation and follow-up in children undergoing bone marrow transplantation (BMT) for MIOP found improvement was apparent within 2 months of successful engraftment with almost complete resolution of abnormalities after 1 year.

Hematopoietic stem cell transplantation for osteopetrosis.

  • C. Steward
  • Medicine
    Pediatric clinics of North America
  • 2010

Hematopoietic stem cell transplantation for infantile osteopetrosis

TLDR
It is recommended early referral for consideration of HCT with a related or unrelated donor as neurosensory manifestations of osteopetrosis are generally not reversible and donor engraftment may be easier to achieve early in the course of the disease.

Successful unrelated mismatched cord blood transplantation in a child with malignant infantile osteopetrosis

TLDR
A case of successful engraftment and stable full‐donor chimerism in a patient with MIOP who underwent unrelated donor cord blood transplantation (CBT) suggests that unrelated donor CBT may be a feasible option in case of unavailability of a fully HLA‐matched related or unrelated donor.

Bone Marrow Transplantation for Infantile Malignant Osteopetrosis

TLDR
BMT offers cure to patients with malignant osteopetrosis with reconstitution of bone marrow and correction of metabolic disturbances with reversibility in neurosensory deficit when BMT is done at an early age.

Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report

TLDR
HSCT is the only curative treatment for autosomal recessive osteopetrosis and should be offered as early as possible in children transplanted before the age of 3 months.

Towards a better understanding and new therapeutics of osteopetrosis

TLDR
HSC‐targeted gene therapy in a mouse model of infantile malignant osteopetrosis was recently shown to correct many aspects of the disease and opens up the possibility for gene replacement therapy as an alternative.

Infantile Malignant, Autosomal Recessive Osteopetrosis: The Rich and The Poor

TLDR
RANKL-dependent patients, in particular, represent an interesting subset which could benefit from mesenchymal cell transplant and/or administration of soluble RANKL cytokine, as expected from biochemical, cellular, and animal studies.

CLINICAL MANIFESTATIONS OF GRAFT‐VERSUS-HOST DISEASE IN HUMAN RECIPIENTS OF MARROW FROM HL‐A-MATCHED SIBLING DONOR,S

TLDR
The results show that despite histocompatibility matching and methotrexate therapy, GVHD remains a serious and often fatal complication of marrow transplantation.