Alternate donor HSCT for thalassemia major from a matched unrelated donor or haploidentical family donor is a feasible therapeutic option in children with no matched family donor. Aggressive pretransplant immunosuppression, reduced toxicity conditioning, and PTCY result in excellent thalassemia‐free survival. We describe here our experience in this cohort. We performed a retrospective analysis of the data on children who underwent a haploidentical HSCT for thalassemia major with PTCY at our center from August 2017 to August 2019. All children received pretransplant immune suppression for 6 weeks with fludarabine and dexamethasone, hypertransfusion and chelation with intravenous desferrioxamine. Conditioning included thiotepa, fludarabine, rabbit ATG, and cyclophosphamide, and GvHD prophylaxis included PTCY with tacrolimus. Twenty children were included and nineteen children engrafted. Acute hypertension occurred in five children, bacterial infection in eight children and viral respiratory infection in three children. Three children suffered from graft rejection. Reactivation of viruses namely CMV, adenovirus, and BK virus was seen in 60% of children. Grades 1‐2 acute GvHD of the skin in four children (20%) and limited chronic GvHD of the skin in four children (20%). Immune cytopenia was documented in three children (15%). Haploidentical HSCT offers a therapeutic option for children with thalassemia major with no suitably matched family or unrelated donors. Our reduced toxicity regimen with PTCY offers a DFS of 75% and OS of 95% with low transplant‐related mortality of 5%.
required PPC services while 58% of them were referred for Physiotherapy services. Those not referred were primarily more stable for short-PICU-stay monitoring. The highest referrals (91.2%) were for respiratory and pain management while early mobilization was the 2nd-highest referral (75.4%), followed by the services for seating and ambulatory equipment prescription (51%). Personalized family support accounted for 35.1% of the referral, in which multidisciplinary pre-discharge plan and caregivers' empowerment program including home respiratory care, strategies for symptomatic relief. Co-jointed home visits were carried out to facilitate safe home discharge. Physiotherapists also provided support for early community integration e.g., outdoor visits.
CONCLUSIONS:This review presents a comprehensive multidisciplinary PPR for patients receiving PPC services in PICU. The teamwork successfully initiates the steps for structured PPR, aiming at providing both children and their family a meaningful journey.
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