If the thalassemia child doesn’t have an HLA-matched compatible donor from the family or from a bone marrow donor bank,there is another cure called Bone Marrow Transplant(BMT) from haploidentical mother to child using protocol 30 (mismatched donor),in which the donor is the mother.
Dr. Pietro Sodani started in 2002 with protocol 30,and in Italy up to December 2011 he performed 43 BMT from haploidentical mother to child with thalassemia with the following results:thalassemia free (no more transfusions) 58%,rejection 31% and mortality 11%. The best results are with very young children in good shape.
The first 22 BMT (from 2002 up to 2008) have been published on Blood journal on November 2009:
Purified T-depleted,CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia
There is a later update with 31 BMT on Pediatric Reports on June 2011:
T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients.
Gamma delta T cells
In January 2012 Dr. Sodani improved the protocol 30 using a technique called “gamma delta T cells” to better select the stem cells of the mother. The preliminary results (still unpublished) from January 2012 to November 2015 in Italy with 12 children (9 thalassemia e 3 Sickle cell) age 3-14 years are:
- 10 overall survival
- 9 disease free with persistent engraftment
- 1 rejection with autologous reconstitution,but still transfusion free and thalassemia free
- 1 rejection
- 1 mortality
Pietro Sodani’s talk at TEDMEDLive Bologna on BMT from from haploidentical mother to child in thalassemia as a social business.
Background and history
In the 1990’s Prof. Lucarelli started doing haploidentical mismatch transplants using the mother for patients in advanced leukemia. BMT from haploidentical mother to child with thalassemia has been invented in 2002 by Dr. Pietro Sodani (Cure Thalassemia co-founder and scientific coordinator) and Prof. Lucarelli and published on Blood journal on November 2009:
Purified T-depleted,CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia.
When in 2002 Prof. Lucarelli and Dr. Sodani started doing it,they already had 10 years of experience using the mother as haploidentical donor.
The Blood paper of Dr. Sodani has been positively commented on another Blood paper written by Ann Woolfrey,who works in the most famous BMT center in the world,the Fred Hutchinson Cancer Research Center in Seattle:Breaking barriers. Here an excerpt of the abstract:
“Although not perfect,the results are extremely promising—a reasonably small risk for mortality with a reasonably high rate of engraftment. Among patients who did not achieve full donor chimerism,disease symptoms were ameliorated in those with mixed donor-host chimerism and autologous hematopoiesis resumed in those who rejected their graft.
The fact that these results were obtained in a group of mostly class 2 to 3 thalassemia patients is even more remarkable.”
Description of the timeline of the BMT from haploidentical mother to child with thalassemia
Preparation at home + day hospital/outpatient
day 1 Cryopreservation of the bone marrow of the child (autologous)
day 2-59 every day some drugs at home,2 times a week blood transfusions in the hospital
BMT in the hospital
day 60 admitted in the BMT unit
day 71 after 11 days from the admission,there is the bone marrow infusion
day 85 after 20 days from the infusion a bone marrow harvest to see the engraftment
day 95 the child leaves the hospital,if the engranfment is OK and there are no complications
Follow up in day hospital/outpatient
the first 2 months,2 times each week at the hospital
the third month,once a week at the hospital
from the beginning of forth month no need to go to the hospital,just regularly consultation and check for your doctor every 2 weeks.
if possible,after 6 months go again to the hospital to do a bone marrow harvest to check the situation
Ask the free advice of Dr. Sodani (our scientific coordinator) to see if you can have BMT from haploidentical mother,and what the results might be.