Satyendra Katewa

SUCCESSFUL CONTROL OF MASSIVE GASTROINTESTINAL BLEEDING FOLLOWING UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) BY USE OF RECOMBINANT ACTIVATED FACTOR VII (RFVIIA) AND OCTREOTIDE INFUSION

Post hematopoietic stem cell transplantation (HSCT) gastrointestinal (GI) bleeding is a dreaded complication. There are only five other reports (one randomised trial and four case reports) of use of rFVIIa for massive lower GI bleeding post-allogeneic HSCT. In only one publication, two adult patients showed complete response. Eroglu has reported a response rate of 50% to octreotide in gastrointestinal bleeding in patients without portal hypertension. We present a 10 month-old female child, who had three episodes of life threatening lower GI bleeding post unrelated Umbilical Stem Cell Transplant (UCBT) controlled successfully each time by use of rFVIIa and octreotide infusion and review of literature. To our knowledge this is the first and youngest case reported, in which both rFVIIa and octreotide have been used successfully to control life threatening lower GI bleeding post UCBT.

Will Post-Transplantation Cell Therapies for Pediatric Patients Become Standard of Care?

Although allogeneic hematopoietic stem cell transplantation (HSCT) is a curative approach for many pediatric patients with hematologic malignancies and some nonmalignant disorders, some critical obstacles remain to be overcome, including relapse, engraftment failure, graft-versus-host disease (GVHD), and infection. Harnessing the immune system to induce a graft-versus-tumor effect or rapidly restore antiviral immunity through the use of donor lymphocyte infusion (DLI) has been remarkably successful in some settings. Unfortunately, however, the responses to DLI can be variable, and GVHD is common. Thus, manipulations to minimize GVHD while restoring antiviral immunity and enhancing the graft-versus-tumor effect are needed to improve outcomes after allogeneic HSCT. Cellular therapies, defined as treatment modalities in which hematopoietic or nonhematopoietic cells are used as therapeutic agents, offer this promise for improving outcomes post-HSCT. This review presents an overview of the field for pediatric cell therapies in the transplant setting and discusses how we can broaden applicability beyond phase I.

Barriers to Cure for Children with Cancer in India and Strategies to Improve Outcomes: A Report by the Indian Pediatric Hematology Oncology Group

The survival of children with cancer in India is inferior to that of children in high-income countries. The Indian Pediatric Hematology Oncology Group (IPHOG) held a series of online meetings via www.Cure4kids.org to identify barriers to cure and develop strategies to improve outcomes. Five major hurdles were identified: delayed diagnosis, abandonment, sepsis, lack of co-operative groups, and relapse. Development of regional networks like IPHOG has allowed rapid identification of local causes of treatment failure for children with cancer in India and identification of strategies likely to improve care and outcomes in the participating centers. Next steps will include interventions to raise community awareness of childhood cancer, promote early diagnosis and referral, and reduce abandonment and toxic death at each center. Starting of fellowship programs in pediatric hemato-oncology, short training programs for pediatricians, publishing outcome data, formation of parent and patient support groups, choosing the right and effective treatment protocol, and setting up of bone marrow transplant services are some of the effective steps taken in the last decade, which needs to be supported further.

Successful Reduced Intensity Conditioning Alternate Donor Stem Cell Transplant for Wiskott-Aldrich Syndrome

There are very few reports of reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) with alternate donor for Wiskott-Aldrich syndrome (WAS) and there is no report of RIC with posttransplant cyclophosphamide (PTCy) in WAS. There is only 1 report of T cell receptor &agr;&bgr; and CD19-depleted haploidentical HSCT for WAS. Here we report successful outcome in 3 children with WAS who underwent successful RIC alternate donor HSCT of whom 2 (matched unrelated donor and T-cell replete haploidentical) received PTCy and 1 underwent T cell receptor &agr;&bgr; and CD19-depleted haploidentical HSCT. We modified conditioning used by Luznik for haploidentical HSCT by adding thiotepa 8 mg/kg and Campath or rabbit antithymoglobulin for 2 cases who received PTCy. In third case we gave fludarabine, thiotepa, and treosulfan-based conditioning. The mean duration of follow-up for these patients was 23.6 months posttransplant (range, 21 to 26 mo). All 3 patients are transfusion independent. Acute graft versus host disease (GVHD) grade I occurred in 1 and none had chronic GVHD. Chimerism of all 3 was fully donor (>95% donor) at D+30 and D+100 posttransplant. All are alive, healthy, and doing well. Our 3 cases highlight that with newer conditioning and GVHD prophylaxis approach alternate donor HSCT in WAS can become a safe and effective treatment option.

Reduced-toxicity alternate-donor stem cell transplantation with posttransplant cyclophosphamide for primary immunodeficiency disorders

We describe here the outcomes of reduced‐toxicity alternate‐donor stem cell transplant (SCT) with posttransplant cyclophosphamide (PTCy) for primary immunodeficiency disorders (PIDs) in eight children (haploidentical—seven and matched unrelated donor—one). The conditioning was with serotherapy (alemtuzumab‐3/rabbit‐anti‐thymoglobulin‐5); fludarabine, cyclophosphamide, and total body irradiation‐5 (additional thiotepa‐3); fludarabine and treosulfan‐2; and fludarabine and busulfan‐1. All received PTCy 50 mg/kg on days 3 and 4 as graft versus host disease prophylaxis along with tacrolimus and mycophenolate. Mean CD34 dose was 13.8 × 106/kg. Two children died because of PIDs. Acute graft versus host disease up to grades I and II was seen in three children. All six survivors are fully donor and disease free at median follow‐up of 753 days. Alternate donor SCT with PTCy is feasible in PID and has good outcomes.

Recurrent anemia in an infant with pneumonia: be vigilant for uncommon presentation of cystic fibrosis

Cystic fibrosis (CF) is an autosomal recessive multisystem disorder characterized by the formation of thick, sticky mucus that can hamper our various organs.1 CF is caused by the alteration of a gene located on the long arm of chromosome7 that encodes a protein, the cystic fibrosis trans membrane conductance regulator (CFTR), which functions as a chloride channel on the apical membrane of epithelial cells.2 The disorder’s common symptomatology is due to progressive damage to the respiratory system, digestive system, exocrine pancreatic insufficiency (diarrhea and failure to thrive), though their severity varies among affected individual.3 Those infant who are not having classical clinical manifestations suggestive of CF or uncommon presentation, neonatal screening or early suspicion is helpful in detection of disease which allows prompt treatment of CF related complications, improving survival and conceptualizing the treatment strategies.4 Meconium ileus may be the first manifestation of CF in the neonatal period, occurring in approximately 20% of patients with pancreatic insufficiency.5 Though iron deficiency anemia may be present due to malabsorption in children,6 transfusion dependent severe anemia in early infancy associated with CF is not mentioned in literature. Our objective of the present publication is to report the case of an infant who had uncommon presentation of CF with recurrent anemia and pneumonia without full blown evolution of the disease despite the clinical suspicion of CF.

MIBG followed by haploidentical stem cell transplant with post transplant cyclophosphamide in relapsed/refractory neuroblastoma.

e22002Background: Relapsed/Refractory Neuroblastoma(RR-NB) outcomes are dismal. One study of Iodine-131-labled MIBG (131I-MIBG) and T-cell depleted haploidentical Stem Cell Transplant (SCT) has sho...