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Submitted by PatientsEngage on 28 June 2016

Texan Chuck Dandridge became the first adult in the U.S. to receive a newly modified stem cell transplant that uses genetically engineered blood cells from a family member. This was announced by researchers at UT Southwestern Medical Center’s Harold C. Simmons Comprehensive Cancer Center where the procedure was performed.

PatientsEngage interviewed Dr. Madhuri Vusirikala, Director of UT Southwestern’s National Marrow Donor Program, part of the stem cell transplant program. 
 

1.  Can all leukemia patients be eligible for stem cell transplant treatment?
At some level yes. That is a difficult question to answer as it depends on the type of leukemia (there is no need to transplant some leukemias as they are highly curable with chemo or targeted agents-eg. Chronic myeloid leukemia, acute promyelocytic leukemia) AND the patient (some patients with multiple medical issues, those who are elderly and debilitated are not candidates for transplant)
 
2.  What other cancers can stem cell transplant be used for?
Myeloma
Lymphoma
Testicular cancer
Many other hematological disorders such as aplastic anemia, thalassemia, sickle cell disease, myelodysplasia, myelofibrosis etc. Lot of researchers are considering transplant for conditions such as lupus.
 
3.  What is the eligibility criterion for stem cell transplant?
Depends on type of transplant, autologous (using one's own stem cells) versus allogeneic (using a donor- related, unrelated, cord blood)
Typically we say patient eligibility criteria is as follows:

  • They have a disease that would benefit from a transplant
  • The patient is in a good condition to tolerate transplant (good organ function, no active infections, good physical condition etc)
  • If a donor needed, the donor is in a good condition and able to donate.

4.  In case of no immediate blood relative, what are the chances of finding a donor from the minority population? How about someone of Indian or Chinese or Malay origin?
Difficult. Unfortunately, there is no stem cell registry in India. So, if one of the patients from India needs an unrelated donor, they search in the American registry as there are several Indians registered here. Minorities (Hispanic, Asians, African americans) represent only 20-30% of the donor registry!
Newer technology is allowing us to transplant patients using half matched donors safely(so, one’s parent or child can serve as a donor and this has widened donor choices)
 
5.  Is this treatment available in India or in South East countries Singapore or Malaysia?
I know it is available in India in several centers: AIIMS, CMC Vellore, Tata Memorial for sure.
I am not aware of centers in Singapore and Malaysia. Thailand has a robust transplant program. In fact, I have trained a couple of Thai docs who are back there and practicing.
 
6.   What is the cost of such a treatment?
I am not sure what the costs in India are but have heard numbers like Rs. 10 lacs per transplant. Lot will depend on long term complications (especially in an allogeneic transplant). When we get patients who are self pay, for autologous transplant, we charge them USD 100,000 and for allogeneic transplant, USD 250,000.
 
7.   What is the prognosis for most patients post treatment?
Again depends on the disease. Transplant is palliative (non curative) for certain diseases such as myeloma, lupus and curative for those such as leukemia. The type of leukemia, type of donor, state of the disease at transplant all determine outcomes. Most of the transplant outcomes are quoted as survival and mortality rates.
For example: 100 day mortality rate for autologous transplant in US is 1% and that for related allogeneic transplant is 5-6%. Survival rates depend on type of disease.
 
8.   What are the side-effects of stem cell transplant?
The side effects of transplant depend on the type of transplant.

  • The side effects are related to chemotherapy used for the transplant, depends on the type of chemo used
  • The side effects can be related to the low blood counts after the chemo and immunocompromised state which result in infections, need for transfusions, fatigue etc.
  • Side effects due to donor cells (graft versus host disease) in the allogeneic setting. This can result in need for long term anti-rejection medications.

 9.  Anything you would like to add for our community.
 
This is such a wide and evolving field, I think further information for your readers can be provided on a case by case basis. 
Take home message is that several cancers are curable, stem cell transplant is very much feasible and the outcomes are improving by the day!

 

Dr. Madhuri Vusirikala is also Professor of Internal Medicine and the primary investigator of many UT Southwestern clinical trials related to bone marrow transplantation.  For more information of the stem cell transplant in the U.S, read here 

 

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