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1.
Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation
Missontsa, M. M., Bernaudin, F., Fortin, A., Dhédin, N., Pondarré, C., Yakouben, K., Neven, B., Castelle, M., Cavazzana, M., Lezeau, H., et al
Journal of assisted reproduction and genetics. 2024
Abstract
PURPOSE To describe the experience of performing ovarian tissue cryopreservation (OTC) before hematopoietic stem cell transplantation (HSCT), among girls/women with severe sickle cell disease (SCD)(SS or S/β(0)-thalassemia) who are, besides the usual surgical risk, at risk of SCD-related complications during the fertility preservation procedure for improving their counseling and management. METHODS This retrospective study included 75 patients (girls/women) with SCD who have had OTC before myeloablative conditioning regimen (MAC) for HSCT. Characteristics of patients and data on OTC, ovarian status follow-up, and results of ovarian tissue transplantation (OTT) were collected in medical records. RESULTS At OTC, the median (IQR 25-75; range) age of the patients was 9.6 (6.9-14.1; 3.6-28.3) years, 56/75 were prepubertal, and no SCD or surgery-related complications occurred. The median follow-up post-HSCT was > 9 years. At the last follow-up, among prepubertal patients at HSCT, 26/56 were ≥ 15 years old and presented with a premature ovarian insufficiency (POI), except 2, including the patient who had received an OTT to induce puberty. Eight were 13-15 years old and presented for POI. The remaining 22 patients were under 13. Among the 19 patients who were menarche at HSCT, 2 died 6 months post-HSCT and we do not have ovarian function follow-up for the other 2 patients. All the remaining patients (n = 15) had POI. Five patients had OTT. All had a return of ovarian function. One patient gave birth to a healthy baby. CONCLUSION OTC is a safe fertility preservation technique and could be offered before MAC independent of the patient's age.
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2.
Hoping for a normal life: Decision-making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers
Mekelenkamp, H., de Vries, M., Saalmink, I., Nur, E., Kerkhoffs, J. L., Heijboer, H., Cnossen, M., Lankester, A., Smiers, F.
Pediatric blood & cancer. 2023;:e30808
Abstract
BACKGROUND To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation (HSCT). PROCEDURE A qualitative longitudinal multicenter study. Data collection consisted of 40 audio-recorded conversations between physicians and families and 77 interviews with patients and/or caregivers related to 27 unique cases, collected at different time points throughout the decision-making process. RESULTS Conversations and interviews revealed "hoping for a normal life" as an overarching theme, consisting of four main topics: (i) "Building a frame of reference" refers to a process where patients or families try to obtain comprehensive information on HSCT and translate this to their situation to decide. (ii) "Balancing between loss and benefit" reports the process of considering the advantages and disadvantages of continuing with supportive care to treat their disease versus choosing HSCT. (iii) "Experiencing the impact of HSCT" describes the impactfull experience of the HSCT period by those who chose HSCT. (iv) "Balancing again" refers to reflecting on the decision made. CONCLUSIONS The hope for a normal life guided the decision-making process, described as a constant balance between the impact of the disease and HSCT. A structured approach to explore patients' and caregivers' perspectives on HSCT decision-making is needed, where specifically discussing the impact of the disease and hope for a normal life need to be integrated in the process.
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3.
Pre-transplantation vitamin D deficiency increases acute graft-versus-host disease after hematopoietic stem cell transplantation in Thalassemia Major patients
Daloğlu, H., Uygun, V., Öztürkmen, S., Yalçın, K., Karasu, G., Yeşilipek, A.
Clinical transplantation. 2022;:e14874
Abstract
Although there are many studies on the role of vitamin D deficiency (VDD) in hematopoetic stem cell transplantation (HSCT), outcomes have often reported conflicting results because of the heterogeneity of the patients in the studies. We investigated the association between VDD prior to HSCT and outcomes after HSCT in a relatively homogenous group of patients with thalassemia major (TM) who received identical treatment for TM before transplantation, and the same conditioning regimen and GVHD prophylaxis during and after transplantation. All patients, including the patients with normal vitamin D 3 levels received 400 to 800 IU per day of vitamin D for the first six months after HSCT. Pre-HSCT VDD increased the frequency of aGVHD after transplantation, particularly in HSCTs performed with PBSC for the stem cell source. Pre-transplant low vitamin D 3 levels had no association with transplant outcomes such as engraftment, viral infections, alloimmunization, chronic GvHD, total days of hospitalization, and success in terms of transfusion independence. Low vitamin D 3 levels before HSCT carry a significant risk for aGVHD. All patients with TM should be screened for VDD before HSCT, and every effort should be made to supplement vitamin D before the transplant in VDD patients. This article is protected by copyright. All rights reserved.
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4.
Granulocyte Colony-Stimulating Factor is Safe and Well Tolerated following Allogeneic Transplantation in Patients with Sickle Cell Disease
Shah, N. C., Bhoopatiraju, S., Abraham, A., Anderson, E., Andreansky, M., Bhatia, M., Chaudhury, S., Cuvelier, G. D. E., Godder, K., Grimley, M., et al
Transplantation and cellular therapy. 2021
Abstract
Granulocyte colony-stimulating factor (G-CSF) used after hematopoietic stem cell transplantation (HSCT) can enhance neutrophil recovery in patients rendered neutropenic by the preparative regimen. G-CSF is contraindicated in patients with sickle cell disease (SCD) as life-threatening complications can ensue in the presence of sickle vasculopathy. However, the safety profile of G-CSF after HSCT for SCD has not been previously described. We report clinical outcomes in the first 100 days post-HSCT in patients supported with G-CSF until neutrophil recovery on a clinical trial of reduced intensity transplantation for SCD. Patients (n=62) received G-CSF for a median of 9 days (range, 5-33) following transplant from the best available stem cell source. Preparation for transplant included a target hemoglobin S level of ≤45%. Neutrophil engraftment (ANC >0.5 × 10(3)/mL) was achieved at a median of 13 days (range,10-34) and platelet engraftment (>50 × 10(3)/mL) at a median of 19 days (range, 12-71). The median duration of inpatient hospitalization following stem cell infusion (day 0) was 21.5 days (range 11-33). No patient developed SCD related complications following G-CSF use. The most common organ toxicities encountered between G-CSF commencement (on day +7) and day +100 were anorexia (14), hypertension (11) and electrolyte imbalance requiring correction (9). Central nervous system related events were noted in 5 patients, all with pre-existing cerebral vasculopathy/moyamoya disease and attributed to reversible posterior leukoencephalopathy syndrome (RPLS) in the presence of calcineurin inhibitor therapy and hypertension. We conclude that G-CSF does not adversely impact SCD transplant recipients and can be safely used post-HSCT to enhance neutrophil recovery.
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5.
Sleep disturbance in adults with sickle cell disease: relationships with executive and psychological functioning
Rhodes, A., Martin, S., Wolters, P., Rodriguez, Y., Toledo-Tamula, M. A., Struemph, K., Fitzhugh, C., Hsieh, M., Tisdale, J.
Annals of hematology. 2020
Abstract
Sleep disturbance is common among children with sickle cell disease (SCD) and is related to neurocognitive difficulties. However, research on sleep disturbances and related variables among adults with SCD is extremely limited. The present study examined the relationship between sleep, executive functioning, and emotional functioning among 62 adults (29 females; M age = 32 years, SD = 7.79) with SCD preparing to undergo a stem cell transplant. Participants were administered a neurocognitive evaluation that included objective and subjective measures of executive functioning, and they completed PROMIS self-report measures of anxiety, depression, and pain intensity. Results showed that about 17% of participants endorsed clinically significant sleep disruptions, while 16.1% and 8% endorsed clinically significant symptoms of anxiety and depression, respectively. Sleep disturbance in these adults was not significantly correlated with objective or subjective measures of executive functioning. Moreover, anxiety, but not depression, was a significant mediator between self-reported sleep difficulties and both objective and subjective measures of executive functioning while controlling for pain intensity. Future research on sleep interventions will be essential for ameliorating the effects of sleep disturbance on executive functioning and anxiety among adults with SCD.
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6.
Hematopoietic stem cell transplantation in children with sickle cell anemia: The parents' experience
Cavadini, R., Drain, E., Bernaudin, F., D'Autume, C., Giannica, D., Giraud, F., Baubet, T., Taieb, O.
Pediatric transplantation. 2019;:e13376
Abstract
Genoidentical HSCT is currently the only curative treatment for SCA, preventing further vascular complications in high-risk children. Studies on the psychological implications of HSCT for recipient, sibling donor, and the rest of the family have been limited in SCA. This study enrolled ten families and used semi-structured interviews to explore the parents' experience at three time points: first before transplantation, then 3 months later, and 1 year later. Three themes emerged from the results: (a) the presence of anxiety, experienced throughout the process, and alleviated by coping strategies (positive thinking, family support, praying); (b) the ability to remain parents to recipient and other family members, despite apprehension and feelings of helplessness, reinforced by the mobilization of important resources at the individual/family levels; (c) the ability to acknowledge the opportunity for their child to be cured of the disease, despite feelings of guilt toward families without a donor, or their own families back home. Overall, the parental experience with HSCT is complex, involving intra-psychic, familial, cultural, religious, and existential factors. Thus, it is important for medical teams to be cognizant of these issues in order to provide the best support to families during the HSCT process.
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7.
Effects of stem cell transplantation on bone mineral density and vitamin D status in children with thalassemia major
Gurlek Gokcebay, D., Ozbek, N., Yazal Erdem, A., Culha, V., Yarali, N., Isik, P., Avci, Z., Azik, F., Demirel, F., Tunc, B.
Pediatric Transplantation. 2017
Abstract
HSCT is a curative treatment in TM, but conditioning and immunosuppressive treatment may affect bone metabolism. In this retrospective study, we aimed to compare BMD, vitamin D status, and growth in children with TM who underwent HSCT to those in children with TD TM. Twenty-three children with TM who underwent HSCT (mean age 7.1 years [1.03-14.7]) and 24 children with TD thalassemia (mean age 9.8 years [1.6-14]) were recruited. Lumbar spine BMD of TD thalassemia patients was higher than those in patients who had HSCT at both baseline and second-year assessments (P=.009, P<.001, respectively). However, BMD Z scores or serum 25-OH vitamin D levels were not different in two groups. Being >10 years of age was a significant risk factor for low BMD, height, and weight Z score for both groups. Patients who underwent HSCT with Pesaro risk class II or III had higher risk for low BMD compared to those risk class I patients (P=.044). In conclusion, children with TM who were >10 years at HSCT are at risk for low BMD and growth retardation. HSCT had no effect on BMD deficit in children with TM.Copyright © 2017 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.