A 10-Year Follow-up Study of Children With Thalassemia Major Post-transplantation Using Treosulfan, Thiotepa, and Fludarabine-Based Conditioning Regimen and Its Impact on Growth and Puberty.

Background: We present a uniform cohort of children with thalassemia major who underwent treosulfan-based conditioning for hematopoietic stem cell transplantation (HSCT) and its impact on growth and puberty.

Methods: The study included retrospective analysis of children up to 18 years of age who underwent allogeneic HSCT for transfusion-dependent thalassemia major between 2010 to 2020, with a minimum follow-up period of 2 years.

Results: Of 202 children in the study, 59% were male and 41% were female; 110/202 (54%) had a matched family donor (MFD), 62/202 (31%) had a haploidentical donor, and 30/202 (15%) had a matched unrelated donor (MUD).

Patterns of Care and Survival of Wilms Tumor in Children in India: A Retrospective Multicentric INPHOG Study.

Unlabelled: Renal cancers are rare in children, accounting 6-7% of childhood tumors. In India, there is paucity of data on renal tumors including Wilms tumor (WT).

Aims: To describe the patterns of care of children with WT; 1, 3, and 5 year event-free survival (EFS) and overall survival (OS).

Allogeneic hematopoietic stem cell transplantation for STAT3 hyper-IgE syndrome: a worldwide study.

Signal transduction and activator of transcription 3 hyperimmunoglobulin E syndrome (STAT3-HIES) is a multisystem disorder causing recurrent skin and respiratory infection with bronchiectasis, pneumatoceles, and aspergillosis; lymphoma; and extraimmune manifestations including fractures and vasculopathy. Published data on immune and extraimmune hematopoietic stem cell transplant (HSCT) outcomes focus on case reports or small cohorts. We conducted an international multicenter retrospective study of HSCT in STAT3-HIES.

Risk Stratification-Based Therapy for Children with E-beta Thalassemia: A 20-Year Follow-Up Study.

Objective: To present risk stratification-based follow-up of children with E-beta thalassemia.

Methods: Children with E-beta thalassemia between August 2002 and August 2022 were classified as mild, moderate, or severe disease using Mahidol scoring. Children in the moderate group with < 20 or ≥ 20 nucleated red blood cells (nRBCs) per 100 white blood cells were clubbed with mild or severe phenotype, respectively.

Haploidentical stem cell transplantation in newly diagnosed high-risk neuroblastoma: Results from a single-arm, prospective study from India.

Background: With increasing data on graft-versus-tumor (GVT) effect in neuroblastoma, we aimed to evaluate the possibility of haploidentical stem cell transplantation (HSCT) as a technique to induce GVT and thereby improve outcomes in high-risk neuroblastoma.

Patients And Methods: We performed a prospective, single-arm study and included children from 18 months to 18 years of age diagnosed with high-risk neuroblastoma and who underwent a haploidentical HSCT from a parent donor. All children were started on induction chemotherapy as per the SIOP-Europa-Neuroblastoma (SIOPEN) protocol, followed by assessment and surgery when feasible.

Integrated Pediatric Intensive Care and Hematopoietic Stem Cell Transplantation Service Improves The Peri-Transplant Survival In Children.

Background: Peri-transplant is a critical period which is associated with a myriad of complications that require pediatric intensive care unit (PICU) referral. PICU outcomes have been historically poor post-hematopoietic stem cell transplantation (HSCT), especially when associated with inotrope support, invasive ventilation, and renal replacement therapy. The study aimed to assess the outcomes of PICU referral in children undergoing HSCT.

Haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for Fanconi anemia with/without anti-thymocyte globulin.

Background: We present comparative data of children with Fanconi anemia undergoing haploidentical hematopoietic stem cell transplantation (HSCT) with or without the addition of rabbit anti-thymocyte globulin (r-ATG) to the conditioning regimen.

Patients And Methods: This retrospective study included children with Fanconi anemia aged up to 18 years who underwent haploidentical HSCT between January 2015 and December 2022. The children were included in two cohorts in this study.

Real-World Data on Childhood Relapsed Acute Lymphoblastic Leukemia: A Report on 100 Children Over two Decades From Southern India.

Objective: The present study aims to provide outcome data in children with relapsed acute lymphoblastic leukemia (ALL) over two decades and variables that impact survival.

Method: This retrospective study included children who were diagnosed with ALL and treated at our center and relapsed between March 2002 and March 2021.

Results: A total of 100 children (64 boys, 36 girls) were included; 80 had B-ALL, 20 had T-ALL.

Evaluation of Pathway to Diagnosis of Pediatric Brain Tumors in Tamil Nadu, India.

Purpose: Delayed diagnosis and poor awareness are significant barriers to the early intervention of pediatric brain tumors. This multicenter observational study aimed to evaluate the baseline routes and time to diagnosis for pediatric brain tumors in Tamil Nadu (TN), with the goal of promoting early diagnosis and timely referrals in the future.

Methods: A standard proforma was used to retrospectively collect information on demographics, diagnosis, referral pathways, and symptoms of incident pediatric brain tumor cases between January 2018 and October 2020 across eight tertiary hospitals in TN.

Cytomegalovirus Reactivation as a Risk Factor for All-Cause Mortality in Children Undergoing Hematopoietic Stem Cell Transplantation: Experience Over Two Decades from a Tertiary Referral Center in India.

The aim of the study was to analyse the burden of cytomegalovirus (CMV) disease in children undergoing hematopoietic stem cell transplantation (HSCT) and its correlation with all-cause mortality. We performed a retrospective study in children up to 18 years of age who underwent allogeneic HSCT between February 2002 to December 2021 in the pediatric blood and marrow transplantation unit. A total of 1035 patients were included where five hundred forty-three (52.

Deciphering stage 0 hematogones by flow cytometry in follow-up bone marrow samples of pediatric B-Acute lymphoblastic leukemia cases: A potential mimicker of residual disease after anti CD19 therapy.

CD19 is frequently targeted for immunotherapy in B cell malignancies, which may result in loss of CD19 expression in leukemic cells as an escape mechanism. Stage 0 hematogones (Hgs) are normal CD19-negative very early B cell precursors that can be potentially mistaken for CD19 negative residual leukemic cells by flow cytometry (FCM) in B cell acute lymphoblastic leukemia (BCP-ALL) cases treated with anti CD19 therapy. Our main objective was to characterize and study the incidence of stage 0 hematogones in follow-up bone marrow samples of pediatric BCP-ALL cases.

Genital Graft-versus-Host Disease in Children: An Underdiagnosed and Undertreated Entity.

Genital graft-versus-host disease (GVHD) is an underdiagnosed and poorly recognized complication, especially in the pediatric population. We report our data on children with genital manifestations of GVHD and their unique clinical features. The study included children up to age 18 years who underwent hematopoietic stem cell transplantation (HSCT) over a 20-year period from February 2002 to February 2022.

An International Survey of Allogeneic Hematopoietic Cell Transplantation for X-Linked Agammaglobulinemia.

Purpose: X-linked agammaglobulinemia (XLA) is an inborn error of immunity caused by variants in Bruton's tyrosine kinase (BTK). XLA patients require lifelong immunoglobulin replacement therapy (IgRT). Only few XLA patients are indicated for allogeneic hematopoietic cell transplantation (HCT) because of severe complications.

Risk Factors for Neutropenic Sepsis Related Mortality in Children Undergoing Allogenic Hematopoietic Stem Cell Transplantation.

We aimed to analyze infections in children undergoing hematopoietic stem cell transplantation (HSCT) until engraftment. The spectrum and risk factors associated will help plan interventions to reduce mortality. We performed a retrospective analysis on the infections, associated risk factors, and mortality until engraftment in children up to 18 years of age undergoing HSCT from January 2017 to August 2020.

Impact of Conditioning Regimen and Graft-versus-Host Disease Prophylaxis on The Outcome of Haploidentical Peripheral Blood Stem Cell Transplantation for High-Risk Severe Aplastic Anemia in Children and Young Adults: A Report from the Pediatric Severe Aplastic Anemia Consortium of India.

Allogenic hematopoietic cell transplantation (HCT) is the best curative approach for patients with severe aplastic anemia (SAA). The outcomes of HCT from haploidentical family donors (HFDs) have improved, making it a feasible option for patients lacking an HLA-identical donor. However, data on HFD-HCT for younger patients with SAA is sparse.

Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades.

Objective: We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM).

Methods: We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020.

Results: 24 children, (mucopolysaccharidosis - 13, Gaucher disease - 4, X-linked adrenoleukodystrophy - 4, metachromatic leukodystrophy - 2, Krabbe disease - 1) were included.

Donor Characteristics Predict the Success of Allogeneic Hematopoietic Stem Cell Transplantation in Thalassemia Major: A Single-Center Analysis of 250 Patients.

We present data on the impact of donor characteristics in a uniform cohort of children who underwent hematopoietic stem cell transplantation (HSCT) for thalassemia major. We performed a retrospective study in children undergoing matched related (MRD) or unrelated (MUD) HSCT from January 2009 to December 2019. We analyzed data on 250 patients (age seven months-19 years), MRD n = 187, MUD n = 63.

Treosulfan-Based Conditioning in Matched Family, Unrelated and Haploidentical Hematopoietic Stem Cell Transplantation for Genetic Hemophagocytic Lymphohistiocytosis: Experience and Outcomes over 10 Years from India.

We aimed to analyze data in children with primary hemophagocytic lymphohistiocytosis (HLH) who underwent hematopoietic stem cell transplantation (HSCT). We performed a retrospective study where children up to 18 years, with primary HLH and who underwent HSCT from January 2011 to December 2019, were included. Twenty-five children with genetic HLH underwent HSCT, including variants (Griscelli syndrome (GS2) 7, Chediak-Higashi syndrome (CHS) 2, XIAP mutation 2).

Clinical, immunological and genomic characteristics of children with X-linked agammaglobulinemia from Kerala, South India.

X-linked agammaglobulinemia (XLA) is an X-linked recessive primary immunodeficiency disorder caused due to a pathogenic variant in the Bruton tyrosine (BTK) gene with an incidence of 1:379,000 live births and 1:190,000 male births. Patients affected with XLA present with recurrent infections of the gastrointestinal and respiratory tracts. Here we report the first case series of 17 XLA patients of 10 South Indian families with a wide spectrum of clinical and genetic features.