Encouraging Outcomes of Alternate Donor Hematopoietic Stem Cell Transplant in Pediatric High-risk/Relapsed Leukemias: A Single Center Experience.

Outcomes of high-risk and relapsed pediatric acute leukemias continue to be suboptimal. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative modality. However, <30% of patients have matched sibling donors available. Hence, alternate donors (matched unrelated and haploidentical) are being used to improve outcomes. We retrospectively analyzed our data of all children with high-risk/relapsed acute leukemias who underwent alternate donor HSCT at our center from April 2015 to July 2020. A total of 15 patients were included-3 underwent matched unrelated and 12 underwent haploidentical HSCT. Before HSCT, all patients were in complete remission (CR): CR1-1, CR2-11, and CR3-3. All patients engrafted except one. Median time to neutrophil and platelet engraftment was 15 and 16 days, respectively. There were 3 transplant related mortalities. One patient was lost to follow-up. Remaining 11 patients remain in remission and are alive. The cumulative incidence of acute graft versus host disease was 57.1% and of chronic graft versus host disease was 21.4%. Overall survival was 80% and the event-free survival was 73.3%. The median follow-up of alive patients was 775 days (range: 333 to 2077 d). Our experience shows encouraging outcomes using alternate donor HSCT for these patients from developing world.

Allogeneic Hematopoietic Stem Cell Transplant Offer Good Outcomes in Pediatric Aplastic Anemia: Experience From Developing World.

Between 2014 and 2020, 31 patients with severe aplastic anemia (SAA) underwent full match allogeneic hematopoietic stem cell transplantation at our center. Of the 31 patients with SAA, 19 had acquired aplastic anemia, 2 had Diamond Blackfan anemia and 10 had Fanconi anemia. Donors were either matched sibling (n=29), related donors (n=2), or unrelated donors (n=3). Peripheral blood stem cells were the graft source in all the cases except 1. Fludarabine-based reduced intensity conditioning was used in all except for patients with a diagnosis of Diamond Blackfan anemia. All patients except 1 achieved hematologic recovery in the form of neutrophil engraftment at 13 days (range, 9 to 17), whereas platelet engraftment occurred at 14 days (range, 10 to 18). Graft versus host disease (GvHD) prophylaxis consisted of cyclosporine and methotrexate ±antithymocyte globulin (horse/rabbit). Acute GvHD developed in 12.9% patients, whereas no patients developed chronic GvHD till the time of last follow-up. The 2-year overall survival for the entire cohort was 93.21±4.6%. In patients with SAA, allogeneic stem cell transplant using fludarabine-based conditioning regimens are very well tolerated and have excellent outcomes in a full match setting.

Haploidentical Stem Cell Transplant With Post-transplant Cyclophosphamide for Chediak-Higashi Syndrome: A Very Rare Case Report.

Chediak-Higashi syndrome is a rare immunodeficiency disorder for which hematopoietic stem cell transplant (HSCT) is the only curative treatment option. HSCT only corrects the hematologic and immunologic manifestations of the disease but neurologic complications may still progress after transplant. Haploidentical HSCT (haplo-HSCT) has evolved as a feasible alternative for patients with primary immunodeficiency. More recently, there has been use of haplo-HSCT with post-transplant cyclophosphamide. However, only 4 cases of Chediak-Higashi syndrome have been reported using this approach. Here, the authors describe a case of a 17-month-old boy who was successfully treated by haplo-HSCT with reduced-toxicity conditioning (fludarabine/treosulfan/melphalan) and post-transplant cyclophosphamide.

Perception of Indian parents on health-related quality of life of children during maintenance therapy of acute lymphoblastic leukemia: a comparison with siblings and healthy children.

BACKGROUND: Advancements in treatment have improved the prognosis of children with acute lymphoblastic leukemia (ALL). Therefore, there is a need to explore health-related quality of life (HRQOL) in depth, specifically in maintenance therapy, where the available data are minimal. This study was conducted to assess the varied items listed in the domains of HRQOL of Children with ALL during maintenance therapy from a parent's perspective. METHODS: Forty children on the maintenance therapy of ALL, 40 siblings, and 40 healthy children were enrolled, and the HRQOL was assessed by parent proxy reports and child self-reports using PedsQL generic core 4.0 in local language. RESULTS: Parents significantly overrated the HRQOL of ALL patients, their siblings, and healthy children in comparison with child self-report in all domains of health. The HRQOL of children with ALL on maintenance therapy was significantly poorer than siblings and healthy children, but their ability to self-care, household work, attentiveness, and ability to do homework were not affected as per parents' reports. Parents reported that absenteeism because of sickness and hospital visits was more among children with ALL than siblings and healthy children. Children with ALL had emotional problems such as fear, anger, sleeping problems, and worries. In a social health domain, parents reported difficulty in competing among children with ALL. The HRQOL of siblings was as good as healthy children in physical, social, and school health domains as per parents' reports. CONCLUSIONS: In our cohort, parents overrated HRQOL in all groups of children. The study identified the various items in each domain of HRQOL that were affected in children with ALL from parents' perspective; this would guide health care professionals to focus on these specific items so as to improve the overall HRQOL of children with ALL.

Comparison of health-related quality of life of children during maintenance therapy with acute lymphoblastic leukemia versus siblings and healthy children in India.

Data on quality of life (QOL) specifically in maintenance therapy of acute lymphoblastic leukemia (ALL) are minimal. This study was done to assess various items listed in domains of QOL (physical, emotional, social and school health domains) of children with ALL during maintenance therapy, and compare the same with those of their siblings and other healthy children. Forty children on maintenance therapy of ALL, 40 siblings and 40 healthy children were assessed for QOL by child self-report using PedsQL 4.0 Generic Core in the local language. Means were computed and compared for each domain with one-way analysis of variance (ANOVA), wherein higher values reflected better QOL. Overall QOL of children with ALL in maintenance therapy (77.16 ± 10.98) was significantly poorer than that of siblings (93.56 ± 4.41) and healthy children (93.02 ± 3.76) (p < 0.001), but their abilities of self-care, household work, exercise, attentiveness, memory and homework were unaffected. There was significantly higher absenteeism due to sickness and hospital visits, and increased emotional problems (fear, anger, sleeping problems) among children with ALL. In the social health domain, children with ALL reported difficulty in maintaining friendships and competing. QOL of siblings was as good as that of healthy children in physical, social and school health domains, but they had increased emotional problems such as anger and sadness. Healthy children reported significantly higher future worries and bullying than children with ALL and siblings. This study validated that the QOL of children with ALL during maintenance therapy was significantly poorer than that of siblings and healthy children. The study identified various items in each domain of QOL that were affected in these children, and thus would assist in guiding healthcare professionals to focus on these specific items so as to improve their overall QOL.