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Success Story – Kashaf

5 years old, Pancytopenia & hypocellular marrow


Contributed By: Dr. Imtenan Sharif

Five-year-old Kashaf, daughter of a soldier of Pak Army developed complaints of progressive pallor, fatigue and easy bruising in Oct 2014. She had an episode of epistaxis in Feb 2015 for which she was taken to Multan CMH where she was found to have pancytopenia and a hypocellular marrow. In Apr 2015, the patient was referred to AFBMTC for further workup and management. A repeat BM examination revealed 5-10% cellularity, consistent with Very Severe Aplastic Anemia. HLA typing of siblings, parents and first cousins did not reveal a match. Cytogenetics and flowcytometry were negative for Fanconi’s anemia and PNH, respectively. Kashaf was put on Ciclosporin to which there was no response. Considering the disease severity and low probability of response to immunosuppressive therapy, it was decided to take the patient to haplo-identical transplantation. As this was the first patient who was being considered for haploidentical transplant at our center, a new conditioning protocol for aplastic anemia was made. Her donor was 7 years old sister with 75% HLA compatibility. Patient’s and donor’s pre-transplant workup were satisfactory. Pre-transplant conditioning consisted of Flu150Cy100TG10Thiotepa5 with post-transplant Cy100. Kashaf underwent haplo-identical stem cell transplant on 28th Aug 2015. Total nucleated cell dose achieved from primed bone marrow was 16 x108/kg with a CD34 count of 9.12 x106/kg. Neutrophil recovery was on D+14. In addition to post-transplant cyclophosphamide, Ciclosporin and MMF were given for GVHD prophylaxis. Post-transplant, the patient developed hemorrhagic cystitis which she was managed accordingly. She also had CMV reactivation with PCR revealing 11,333 copies/ml and was treated with IV ganciclovir. Her STR for donor chimerism remained more than 90% and lymphocyte subset analysis reported good immune reconstitution at 3, 6 and 12 months respectively. Her immunosuppression was tapered off and stopped after 1 ½ years of transplant. Now, almost 2 years after her transplant, Kashaf has no sign of GVHD and enjoys good quality of life with her family.