Amrith Mathew

Hemophilia treatment center: A stratification model for developing countries: A pilot study from India

Background: Hemophilia center, treatment center (TC), hemophilia TC (HTC), and Hemophilia Comprehensive Care Center (CCC) are terminologies used to describe centers caring for persons with hemophilia (PWH). These are based on their capability to provide multidisciplinary care and laboratory services. Widely described are the European HTCs (EHTCs) and the European Hemophilia CCCs (EHCCCs). However, most centers in developing countries providing care for PWH have variable clinical expertise and laboratory facilities, which do not qualify for the existing models. Materials and Methods: This cross-sectional study was done to evaluate the laboratory and clinical care facilities available in HTCs in India. The survey questionnaire was sent to 62 HTCs in India. Laboratory and clinical care facilities were categorized based on a predefined stratification model. Level IV being the minimum and Level I the maximum were used to define clinical and laboratory facilities. Results: Fifty-two (85%) centers responded representing 17 states in India. Only 28 HTCs had attached laboratory services. Although all the centers cared for acute bleeds, only half managed chronic joint disease (Level III) while one-sixth could perform surgeries (Level II). Only one-third of the laboratories had instituted quality control measures and performed factor assays. Only four centers qualified for EHTC criteria and two for the EHCCC criteria. Conclusion: This HTC stratification model provides assessment and differentiation of the clinical and laboratory services. It allows an individual HTC to identify the standard of care and provides a framework for objectively planning, implementing, and evaluating its services.

Spurious platelet count due to cryoglobulins in a patient with smoldering myeloma.

Use of automated hematology analyzers for routine blood count reporting has increased the reproducibility and accuracy of test results. However, at times, these instruments may generate spurious test results. Such results can result in inappropriate investigations or treatment decisions in patients. Spuriously normal or high platelet counts carry the risk of under diagnosis of the true thrombocytopenia with adverse clinical implications. We present a patient with smoldering myeloma with spurious platelet count due to cryoglobulins.

Unrelated and related donor transplantation for beta-thalassemia major: A single-center experience from India

Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment in patients with β‐thalassemia major. A matched sibling or a related donor is usually found in only 25%‐30% of the patients. There are limited data on matched unrelated donor (MUD) transplants from India. We reviewed HSCT outcome in 56 children with TM who underwent 57 transplants at our center. Related donor (RD) (n=43) and MUD (n=14) transplants were performed with TreoFluT‐based conditioning regimen in majority (95%) of patients. Peripheral blood stem cells (PBSC) were the preferred (85%) source of stem cells. The overall survival (OS) at 1 year in RD and MUD groups was 87.6±5.2% and 85.7±9.4% at a median follow‐up of 25 (1‐92) months and 22.5 (1‐50) months, respectively (P=.757). The thalassemia‐free survival (TFS) at 1 year was 87.6±5.2% and 77.1±11.7% with a median follow‐up of 24 (1‐92) and 16.5 (1‐50) months, respectively (P=.487). Although acute (14% vs 64%) and chronic graft‐versus‐host disease (GVHD) (13.9% vs 42.9%), infectious (39.5% vs 71.4%), and non‐infectious (37.2% vs 78.5%) complications are higher in MUD transplant group, the present data show a comparable OS and TFS among RD and MUD group with treosulfan‐based regimen using PBSC grafts.

Extensive Platelet Phagocytosis in a Patient with Immune Thrombocytopenia (ITP) and Concomitant Tuberculosis

Hemophagocytic syndrome is characterized by benign proliferation of mature histiocytes and uncontrolled phagocytosis of the erythrocytes, leukocytes, platelets and/ or their hematopoietic precursors in the bone marrow. Hemophagocytic syndrome can be familial or acquired which is associated with tuberculosis, autoimmune disorders, hematologic malignancies or solid cancers [1]. Although histiocytes in the bone marrow may show phagocytosis of any of the hematopoietic precursors, marked platelet specific phagocytosis is an extremely rare finding. Inhibitory CD47/(signal regulatory protein) SIRP alpha signaling is involved in regulating platelet phagocytosis in ITP [2]. We present a patient with ITP who had concomitant tuberculosis and showed predominant platelet phagocytosis. A 61 years old male with ITP on steroid therapy presented with fever and cough since 2 weeks. He had pallor with no petechiae or purpuric spots. Investigations showed hemoglobin of 102 g/l with normal white cell count and thrombocytopenia (platelets: 58 9 10/l). Bone marrow aspiration showed increase in histiocytes with many of them showing extensive platelet phagocytosis (Fig. 1). Occasional histiocytes also showed erythroblastic phagocytosis (Fig. 1, Inset). Trephine biopsy and clot section showed epithelioid cell granulomata (Fig. 2a, b). Ziehl–Neelsen stain for acid fast bacilli was positive. HRCT chest was suggestive of military tuberculosis. The Fig. 1 Bone marrow aspirate smear showing extensive platelet phagocytosis by histiocytes. An occasional histiocyte also shows engulfment of erythroblasts [Inset]

Cost Effectiveness of Hematopoietic Stem Cell Transplantation Compared with Transfusion Chelation for Treatment of Thalassemia Major

Hematopoietic stem cell transplantation (HSCT) is the only cure for thalassemia major (TM), which inflicts a significant 1-time cost. Hence, it is important to explore the cost effectiveness of HSCT versus lifelong regular transfusion-chelation (TC) therapy. This study was undertaken to estimate incremental cost per quality-adjusted life-year (QALY) gained with the intervention group HSCT, and the comparator group TC, in TM patients. A combination of decision tree and Markov model was used for analysis. A hospital database, supplemented with a review of published literature, was used to derive input parameters for the model. A lifetime study horizon was used and future costs and consequences were discounted at 3%. Results are presented using societal perspective. Incremental cost per QALY gained with use of HSCT as compared with TC was 64,096 (US

Un-manipulated haploidentical transplant in Wiskott-Aldrich syndrome

986) in case of matched related donor (MRD) and 1,67,657 (US

Macrothrombocytopenia in North India: Role of Automated Platelet Data in the Detection of an Under Diagnosed Entity

2579) in case of a matched unrelated donor transplantation. The probability of MRD transplant to be cost effective at the willingness to pay threshold of Indian per capita gross domestic product is 94%. HSCT is a long-term value for money intervention that is highly cost effective and its long-term clinical and economic benefits outweigh those of TC.

Post stem cell transplantation revaccination: A survey of the current practices in India

BackgroundAllogeneic stem cell transplant is the only curative treatment for Wiskott-Aldrich syndrome.Case characteristics18-months-old boy with no sibling, cord blood or matched unrelated donor transplant options.OutcomeDoing well 7 years after haploidentical stem cell transplantation using unmanipulated bone marrow as the stem cell source.MessageFather as a haplo-identical donor is a feasible option.