Abstract |
Myelodysplastic syndrome (MDS) is often a pernicious disorder associated with pancytopenia in the elderly; therapeutic approaches need to balance their toxicities versus the symptoms of the disease. 1,25(OH)(2)-Vitamin-D(3) [ 1,25(OH)(2)D(3)] inhibits proliferation and induces differentiation of leukemic cells in vitro. Small clinical trials of 1,25(OH)(2)D(3) have shown modest efficacy in MDS; hypercalcemia prevented the administration of doses that have been shown to be effective in vitro. Paricalcitol [19-nor- 1,25(OH)(2)D(2), Zemplar] has been approved by the FDA for treatment of secondary hyperparathyroidism. This Vitamin D analog is unique because it has little hypercalcemic potential; but in vitro, it has strong anti-leukemic activity. We conducted a clinical trial of oral paricalcitol to 12 MDS patients whose disease varied between an IPSS of low to high. Drug was well-tolerated in all patients. No responses were observed according to international working group (IWG) criteria. However, the platelet count of 1 of the 12 individuals rose from 53,500 to 120,000/microl blood over 5 weeks; but the patient succumbed to a fatal fungal infection. In summary, paricalcitol given as a single agent to MDS patients is therapeutically not very efficacious; further trials of the Vitamin D analog should be considered in combination with other approaches.
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Authors | H Phillip Koeffler, Noune Aslanian, James O'Kelly |
Journal | Leukemia research
(Leuk Res)
Vol. 29
Issue 11
Pg. 1259-62
(Nov 2005)
ISSN: 0145-2126 [Print] England |
PMID | 16164982
(Publication Type: Clinical Trial, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)
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Chemical References |
- Ergocalciferols
- paricalcitol
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Topics |
- Administration, Oral
- Aged
- Aged, 80 and over
- Dose-Response Relationship, Drug
- Drug Administration Schedule
- Ergocalciferols
(administration & dosage, therapeutic use)
- Female
- Humans
- Male
- Middle Aged
- Myelodysplastic Syndromes
(diagnosis, drug therapy)
- Treatment Outcome
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