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TOPIC: Umbilical Cord - Stem Cell Treatment - Has serious potential for Refractory ITP

Umbilical Cord - Stem Cell Treatment - Has serious potential for Refractory ITP 5 months 6 days ago #65740

  • jasondema
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Although immune thrombocytopenic purpura (ITP) is generally associated with a good prognosis, some patients do not respond adequately to the existent pharmacological treatments and are exposed to higher risk of severe hemorrhage. The potential of mesenchymal umbilical cord-derived stem cells is being tested to treat this group of ITP patients.

ITP is an autoimmune disorder characterized by an elevated rate of platelet (blood cells responsible for clotting) destruction that occurs in the spleen. Because ITP patients have persistent thrombocytopenia (low blood platelet counts), they are more prone to bleeding. ITP has a relatively broad clinical spectrum. It may be an asymptomatic process in which the patient is essentially followed with no need for medication, or it can become a chronic serious situation in which patients need to undergo splenectomy (surgical removal of the spleen). One of the most common ITP symptoms is the development of petechiae, small-sized purple spots resulting from superficial bleeding in the skin. These can occasionally be accompanied by other symptoms, like bleeding from the gums, blood in urine or unusually heavy menstrual flow.

Umbilical Cord Tissue Stem Cell infusion as an effective treatment for refractory ITP

Chronic refractory ITP patients presenting low platelet counts even after splenectomy and treatment with multiple pharmacological approaches are exposed to a higher risk of severe internal bleeding. The results of an experimental treatment using umbilical cord-derived stem cells to treat 4 ITP patients was recently published. Umbilical cord mesenchymal stem cells have important immunomodulatory properties and have been used in experimental treatments for degenerative and autoimmune diseases. The patients were between 26 and 54 years-old and had persistent ITP that failed to respond to multiple pharmacological therapies. Cord tissue stem cells were administered intravenously and the patients were monitored for 24h, after which they were discharged from the hospital, if no signs of infection or allergic reaction were noted. The procedure was well tolerated and there were no clinically significant side effects observed during the 2-year follow-up period. After the infusion, platelet counts of each patient were measured over time to assess treatment efficacy. Two weeks after administration, all patients had responded favourably to treatment and had higher platelet counts when compared to baseline. One of the patients reached safe platelet counts and was in complete remission for over 2 years after infusion. The other 3 patients relapsed after about 12 months after stem cell treatment and were infused with cord tissue stem cells once again. Response to the second treatment was also positive, with increasing platelet counts observed in all 3 cases.

These results suggest cell therapy to be a viable alternative for treating refractory ITP patients. ITP treatment with umbilical cord tissue-derived stem cells was shown to be safe and effective, even though symptom alleviation was not complete. The authors state that treating chronic refractory ITP patients once a year with cord tissue-derived stem cells might be the best option for these patients.


Wang X et al. Intravenous infusion umbilical cord-derived mesenchymal stem cell in primary immune thrombocytopenia: A two-year follow-up. Exp Ther Med. 2017 May; 13(5):2255-2258.
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Umbilical Cord - Stem Cell Treatment - Has serious potential for Refractory ITP 5 months 5 days ago #65749

  • Hal9000
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Interesting find Jason. One can get the referenced study, in full, here:
Click on 'download' for the PDF.

Capable of full remission. Works on megakaryocytes. Sounds perfect for treating ITP with antibodies against them. Who needs a splenectomy with this option being available?
Based on the characteristics of the evolution of our group of patients, we may emphasize a few important points: i) hUC-MSC transplantation is beneficial for the recovery of bone marrow megakaryocytes, in order that patients can achieve long-term relief; and ii) some cytokines are reduced in this reaction, and the effect of hUC-MSC transplantation is decreased.
I do have to wonder about adverse events. That topic seemed to be skipped.

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Umbilical Cord - Stem Cell Treatment - Has serious potential for Refractory ITP 5 months 3 days ago #65768

  • Sandi
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  • Sandi Forum Moderator Diagnosed in 1998, currently in remission. Diagnosed with Lupus in 2006. Last Count - 344k - 6-9-18
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The downside is that this appears to be a temporary solution for most. I'm not sure about the cost or possible adverse events.

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