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1.
Ann Ist Super Sanita ; 60(2): 107-110, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38984624

RESUMO

AIMS: Using a database from two pharmaceutical companies that managed several compassionate use programs in the last few years in Italy, we have previously analyzed the data by the number of patients and centers in each region and province, showing that the use of compassionate drugs is largely diffused in the country, in a manner directly related to the size of population of each region. In the present study we used the same database to expand the analysis to single-center level, aiming to test the hypothesis whether, despite a good diffusion of compassionate drug uses in each region, the majority of them concentrates within a relatively low number of centers. METHODS: Data from different programs were grouped per center, and the centers were ordered per the number of compassionate uses dispensed, and per region. Two cutoff levels, at 75% and 90%, were drawn to look at the number of centers accounting for such percentages of compassionate uses in each region. RESULTS: Out of 343 centers throughout Italy, 93 and 156 centers (i.e., 27.11% and 45.48% of the total) account for about 75% and 90% of all compassionate drugs dispensed in Italy. In 6 regions out of 20 (Valle d'Aosta, Liguria, Umbria, Lazio, Molise and Campania) the centers accounting for 75% of all compassionate drugs dispensed are located in a single town. Forty and 20 out of the 93 centers dispensing 75% of all compassionate drugs are academic hospitals and research hospitals (Istituti di Ricovero e Cura a Carattere Scientifico, IRCCS), respectively. CONCLUSIONS: In this study we have demonstrated that, in spite of widespread diffusion of compassionate drug uses in all Italian regions, their management is restricted to a relatively low number of dispensing centers in each region.


Assuntos
Ensaios de Uso Compassivo , Itália , Humanos , Bases de Dados Factuais
2.
JPEN J Parenter Enteral Nutr ; 48(5): 624-632, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38837803

RESUMO

BACKGROUND: Selection of central venous catheter (CVC) lock solution impacts catheter mechanical complications and central line-associated bloodstream infections (CLABSIs) in pediatric patients with intestinal failure. Disadvantages of the current clinical standards, heparin and ethanol lock therapy (ELT), led to the discovery of new lock solutions. High-risk pediatric patients with intestinal failure who lost access to ELT during a recent shortage were offered enrollment in a compassionate use trial with 4% tetrasodium EDTA (T-EDTA), a lock solution with antimicrobial, antibiofilm, and antithrombotic properties. METHODS: We performed a descriptive cohort study including 14 high-risk pediatric patients with intestinal failure receiving 4% T-EDTA as a daily catheter lock solution. CVC complications were documented (repairs, occlusions, replacements, and CLABSIs). Complication rates on 4% T-EDTA were compared with baseline rates, during which patients were receiving either heparin or ELT (designated as heparin/ELT). RESULTS: Patients initiated 4% T-EDTA at the time they were enrolled in the compassionate use protocol. Use of 4% T-EDTA resulted in a 50% reduction in CVC complications, compared with baseline rates on heparin/ELT (incidence rate ratio: 0.50; 95% CI, 0.25-1.004; P = 0.051). CONCLUSION: In a compassionate use protocol for high-risk pediatric patients with intestinal failure, the use of 4% T-EDTA reduced composite catheter complications, including those leading to emergency department visits, hospital admissions, additional procedures, and mortality. This outcome suggests 4% T-EDTA has benefits over currently available lock solutions.


Assuntos
Infecções Relacionadas a Cateter , Cateterismo Venoso Central , Cateteres Venosos Centrais , Ácido Edético , Insuficiência Intestinal , Humanos , Estudos Retrospectivos , Ácido Edético/uso terapêutico , Ácido Edético/administração & dosagem , Cateteres Venosos Centrais/efeitos adversos , Feminino , Masculino , Infecções Relacionadas a Cateter/prevenção & controle , Infecções Relacionadas a Cateter/epidemiologia , Pré-Escolar , Lactente , Cateterismo Venoso Central/efeitos adversos , Criança , Heparina/administração & dosagem , Heparina/efeitos adversos , Ensaios de Uso Compassivo , Estudos de Coortes
4.
STAR Protoc ; 5(2): 102949, 2024 Jun 21.
Artigo em Inglês | MEDLINE | ID: mdl-38691464

RESUMO

Phage therapy has re-emerged as a promising treatment for non-resolving infections. Given the lack of approved phage treatments, there is a need to establish a compassionate use pipeline. Here, we present a protocol for phage matching, treatment, and monitoring for compassionate bacteriophage use in non-resolving infections. We describe steps for consultation and request implementation, evaluating and comparing different aspects of phage activity, and phage production. We then detail procedures for multidisciplinary meetings, ethics approvals, phage therapy, and follow-up. For complete details on the use and execution of this protocol, please refer to Onallah et al.1,2.


Assuntos
Bacteriófagos , Ensaios de Uso Compassivo , Terapia por Fagos , Humanos , Bacteriófagos/fisiologia , Terapia por Fagos/métodos , Infecções Bacterianas/terapia
5.
Cancer Med ; 13(10): e7289, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38770551

RESUMO

BACKGROUND: Mantle cell lymphoma (MCL) is a type of B-cell lymphoma that is currently incurable. Pirtobrutinib shows promising response rates in heavily pretreated MCL patients according to the approval study, but the real-world data are scarce. METHODS: In this study, we retrospectively analyzed the efficacy and safety profile of pirtobrutinib in 10 relapsed/refractory MCL patients from compassionate use program (CUP). RESULTS: On average, the patients underwent three lines of systemic therapy prior to pirtobrutinib and were predominantly BTKi exposed (9/10). The best overall response rate (BORR) was 67%. In a median follow-up of 8.6 months, the mean duration of response (DOR), progression-free survival (PFS), and overall survival (OS) were not reached. No new safety signals were documented. CONCLUSIONS: In summary, pirtobrutinib represented a safe and effective treatment option in a small real-world population.


Assuntos
Ensaios de Uso Compassivo , Linfoma de Célula do Manto , Humanos , Linfoma de Célula do Manto/tratamento farmacológico , Masculino , Feminino , Idoso , Pessoa de Meia-Idade , Estudos Retrospectivos , Europa (Continente) , Resultado do Tratamento , Recidiva Local de Neoplasia/tratamento farmacológico , Idoso de 80 Anos ou mais , Inibidores de Proteínas Quinases/uso terapêutico , Inibidores de Proteínas Quinases/efeitos adversos , Intervalo Livre de Progressão , Adulto , Resistencia a Medicamentos Antineoplásicos
6.
Infect Dis (Lond) ; 56(7): 575-580, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38743059

RESUMO

OBJECTIVE: To study the effect of plitidepsin antiviral treatment in immunocompromised COVID-19 patients with underlying haematological malignancies or solid tumours, particularly those who have undergone anti-CD20 therapies. DESIGN: We conducted a retrospective observational study, involving 54 adults treated with plitidepsin on compassionate use as an antiviral drug. Our analysis compared outcomes between patients with solid tumours and those with haematological malignancies, and a cohort of cases treated or not with anti-CD20 monoclonal antibodies. RESULTS: Patients with a history of anti-CD20 therapies showed a prolonged time-to-negative RT-PCR for SARS-CoV-2 infection compared to non-treated patients (33 d (28;75) vs 15 (11;25); p = .002). Similar results were observed in patients with solid tumours in comparison to those with haematological malignancies (13 (10;16) vs 26 (17;50); p < .001). No serious adverse events were documented. CONCLUSIONS: Patients with haematological malignancies appear to be at a heightened risk for delayed SARS-CoV-2 clearance and subsequent clinical complications. These findings support plitidepsin as a well-tolerated treatment in this high-risk group. A phase II clinical trial (NCT05705167) is ongoing to evaluate plitidepsin as an antiviral drug in this population.KEY POINTSHaematological patients face an increased risk for severe COVID-19.Anti-CD20 therapies could increase fatal outcomes in COVID-19 patients.Persistent viral replication is increased in immunocompromised patients.Plitidepsin does not lead to new serious adverse events in immunocompromised patients.


Assuntos
Tratamento Farmacológico da COVID-19 , COVID-19 , Depsipeptídeos , Neoplasias Hematológicas , Neoplasias , Peptídeos Cíclicos , SARS-CoV-2 , Humanos , Masculino , Feminino , Estudos Retrospectivos , Pessoa de Meia-Idade , Neoplasias Hematológicas/tratamento farmacológico , Neoplasias Hematológicas/complicações , Idoso , Depsipeptídeos/uso terapêutico , Depsipeptídeos/efeitos adversos , Neoplasias/tratamento farmacológico , Neoplasias/complicações , Peptídeos Cíclicos/uso terapêutico , Antivirais/uso terapêutico , Resultado do Tratamento , Adulto , Ensaios de Uso Compassivo , Hospedeiro Imunocomprometido , Antígenos CD20/imunologia , Idoso de 80 Anos ou mais
7.
Arch Cardiovasc Dis ; 117(6-7): 450-456, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38677940

RESUMO

In France, mexiletine - a class I antiarrhythmic drug - can be prescribed for the symptomatic treatment of myotonia of the skeletal muscles in adult patients with myotonic dystrophy under a compassionate use programme. Mexiletine is used according to its summary of product characteristics, which describes its use for myotonia treatment in adult patients with non-dystrophic myotonia, a different neuromuscular condition without cardiac involvement. A cardiac assessment is required prior to initiation and throughout treatment due to potential proarrhythmic effects. The presence of conduction system disease, the most common cardiac manifestation of myotonic dystrophy, mandates repeated cardiac evaluations in patients with this condition, and becomes even more important when they are given mexiletine. A group of experts, including three neurologists and five cardiologists from French neuromuscular reference centres, were involved in a task force to develop a treatment algorithm to guide mexiletine use in myotonic dystrophy. The recommendations are based on data from a literature review of the safety of mexiletine-treated patients with myotonic dystrophy, the compassionate use protocol for mexiletine and the personal clinical experience of the experts. The main conclusion of the expert group is that, although existing safety data in mexiletine-treated patients with myotonic dystrophy are reassuring, cardiac assessments should be reinforced in such patients compared with mexiletine-treated patients with non-dystrophic myotonia. This expert opinion to guide mexiletine treatment in patients with myotonic dystrophy should help to reduce the risk of severe adverse events and facilitate interactions between specialists involved in the routine care of patients with myotonic dystrophy.


Assuntos
Mexiletina , Distrofia Miotônica , Adulto , Humanos , Algoritmos , Antiarrítmicos/uso terapêutico , Antiarrítmicos/efeitos adversos , Arritmias Cardíacas/fisiopatologia , Arritmias Cardíacas/diagnóstico , Arritmias Cardíacas/etiologia , Arritmias Cardíacas/induzido quimicamente , Tomada de Decisão Clínica , Ensaios de Uso Compassivo , Consenso , França , Mexiletina/uso terapêutico , Mexiletina/efeitos adversos , Distrofia Miotônica/tratamento farmacológico , Distrofia Miotônica/diagnóstico , Distrofia Miotônica/fisiopatologia , Medição de Risco , Fatores de Risco , Resultado do Tratamento , Bloqueadores do Canal de Sódio Disparado por Voltagem/uso terapêutico , Bloqueadores do Canal de Sódio Disparado por Voltagem/efeitos adversos
9.
Clin Ther ; 46(4): 374-378, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38461122

RESUMO

PURPOSE: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease causing progressive muscle weakness and reducing life expectancy. Risdiplam (Evrysdi; Genentech/F. Hoffmann-La Roche Ltd, Basel, Switzerland) is a drug approved for use in the treatment of patients with SMA. The ongoing global risdiplam Compassionate Use Program (CUP), initiated in November 2019, is the largest CUP in SMA, currently providing access to risdiplam for >2000 patients with type 1 or 2 SMA in 59 countries. Here, the challenges and learnings from the risdiplam CUP are presented. METHODS: Enrolled patients (aged ≥2 months) had type 1 or 2 SMA and no alternative treatment options (ie, they were not medically eligible for approved SMA treatments, were unable to continue their SMA treatment due to medical reasons, were at risk for lack/loss of SMA treatment efficacy, or did not qualify for/had no access to SMA treatment within a clinical trial). Requests were made by the treating physicians via an end-to-end system. FINDINGS: The risdiplam CUP highlighted the importance of collaborating with patient advocacy groups early to learn about patients' perspectives on unmet medical needs, understanding the sometimes-unique nature of local regulations and requirements, and adapting physician- and patient-eligibility criteria. Key learnings were obtained from enrolling patients from low- to middle-income countries and from countries without dedicated Compassionate Use regulations, and from operating the CUP during the coronavirus disease 2019 pandemic. IMPLICATIONS: The risdiplam CUP experience was successful in many ways and may help to design and implement future CUPs in rare diseases, as well as patients living in countries or in circumstances in which access to innovative treatments is a challenge.


Assuntos
Ensaios de Uso Compassivo , Pirimidinas , Atrofias Musculares Espinais da Infância , Humanos , Atrofias Musculares Espinais da Infância/tratamento farmacológico , Pirimidinas/uso terapêutico , Lactente , Pré-Escolar , Masculino , Feminino , Criança , Compostos Azo
10.
Bone Marrow Transplant ; 59(5): 637-646, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38361117

RESUMO

The ruxolitinib compassionate use (CU) program offered ruxolitinib to patients ≥2 years of age with confirmed steroid-resistant acute or chronic graft-versus-host disease (aGvHD and cGvHD, respectively). Data from 1180 patients (n = 775, 370 and 35 with cGvHD, aGvHD, and non-specified GvHD, respectively) were analyzed. Most patients had severe cGvHD (56%) or stage III/IV aGvHD (70%) disease and had previously received corticosteroids ( > 80%); ruxolitinib was requested primarily as a second-/third-line option. Patients <12 and ≥12 years old most often received the recommended ruxolitinib doses (5 mg twice daily [BID] and 10 mg BID, respectively); however, 23% and 30% of ≥12 year olds with cGvHD and aGvHD, respectively, received the lower dose of 5 mg BID. Notably, corticosteroid usage decreased with ruxolitinib treatment; at the initial ruxolitinib request, 81% and 91% of patients with cGvHD and aGvHD, respectively, were receiving corticosteroids whereas at resupply, 62% and 64%, respectively, were receiving corticosteroids. Eighty two percent of evaluable patients with cGvHD had a complete or partial response to treatment and 56% of evaluable patients with aGvHD had a best response of grade 0/I. These findings demonstrate the rapid and positive effects of ruxolitinib in patients with GvHD in a real-world setting.


Assuntos
Ensaios de Uso Compassivo , Doença Enxerto-Hospedeiro , Nitrilas , Pirazóis , Pirimidinas , Humanos , Doença Enxerto-Hospedeiro/tratamento farmacológico , Pirazóis/uso terapêutico , Pirimidinas/uso terapêutico , Masculino , Feminino , Adulto , Adolescente , Criança , Pessoa de Meia-Idade , Idoso , Adulto Jovem , Pré-Escolar , Transplante de Células-Tronco Hematopoéticas
11.
Ann Pharm Fr ; 82(4): 727-738, 2024 Jun.
Artigo em Francês | MEDLINE | ID: mdl-38408723

RESUMO

The reform of derogatory access authorisations (DAs) on 1st July 2021 has distorted the routine of the hospital pharmacists dealing with innovative medicines that are waiting for marketing authorization or approval. There are two distinct categories of DAs: Compassionate Access Authorisations (CAAs) are granted by the French National Agency for the Safety of Medicines (ANSM) while Early Access Authorisations (EAPs) are granted at the request of pharmaceutical companies by the French National Authority for Health (HAS). All AAPs and a majority of the AACs are supported by a Protocol for Therapeutic Use and Data Collection (PTU-DC). The aim of this study is to assess the impact of the reform on pharmacy process one year following its implementation, and to identify the risks related to the new circuits. The working group, composed of three pharmacists carried out an initial assessment of the effects first measured the impact of the reform on medicine processes in DAs. They performed a comparison of the changes in their management methods: 3 months prior to the reform (M0), and 3 (M3) and 12 months (M12) post-reform. Risks analysis was conducted using the Failure Modes, Effects and Criticality Analysis (FMEA) method. The analysis was limited to the process steps specific related to DAs drugs were analyzed. The critical severity of the risk situations identified was rated. A critical hierarchy matrix was used to establish priority actions. The priority actions to be taken were determined using the critical hierarchy matrix. Over the span of one year, the number of DAs in our establishment showed a 31.7% increase, from 41 at M0 to 54 at M12. At M0, the proportion of drugs needed inclusion via a drug-specific digital platform, specific to each drug, stood at 27% (11/41) of drugs at M0 while at M12, it rose to 52% (28/54). The percentage of PTU-DCs therefore increased by a factor of 1.7, rising from 29% (12/41) at M0 to 47% (21/45) at M3 and 60% (32/54) at M12. For orders, which are always nominative, approval depends on both the presence of the PTU-DC tracking sheet being present in 12% of PAAs, and the inclusion number in 26% of PAAs. The risk analysis shows 49 failure modes leading to risk situations. Among the failure modes, 36 have a consequence of acceptable or tolerable criticality under control, whilst 13 are deemed of unacceptable criticality. A suitable control method exists has been identifies for 5 of them. Finally, the ranking evaluation of criticalities has highlighted 4 situations which require immediate action as a priority: delivery times, obtaining completed tracking sheets and ordering procedures. The aim of the DAs reform is to simplify access to innovative medicines. However, the reform has significant and damaging repercussions on pharmaceutical activities. Corrective measures need to be taken in conjunction with all parties involved in the circuits including laboratories and service providers (CROs), authorities and healthcare professionals.


Assuntos
Farmacêuticos , Serviço de Farmácia Hospitalar , Carga de Trabalho , França , Humanos , Serviço de Farmácia Hospitalar/organização & administração , Medição de Risco , Ensaios de Uso Compassivo , Aprovação de Drogas
12.
Cancer Biother Radiopharm ; 39(5): 323-329, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38324047

RESUMO

Patients with relapsed or refractory metastatic cancer unresponsive to standard therapies have motivated nuclear physicians to develop innovative radioligands, precisely targeted to tumor molecular receptors, for effective treatment of specific advanced malignancies. Individual practitioners in departments of nuclear medicine across the world have performed first-in-human studies on compassionate patient usage N-of-One protocols. These physician-sponsored studies then evolved into early-phase clinical trials and obtained real-world data to demonstrate real-world evidence of effectiveness in prolonging survival and enhancing quality of life of many so-called "End-Stage" cancer patients. Virtually all the therapeutic radiopharmaceuticals in current clinical oncology have been discovered and developed into effective specific treatments of targetable cancers by individual doctors in the course of their hospital practice. Pharma industry was not involved until many years later when performance of mandated Phase 3 randomized controlled trials became necessary to achieve regulatory agency approval. This article traces the history of several novel theranostic agents developed from compassionate N-of-One studies by hospital physicians over the past 36 years. It acknowledges the collegiality and collaboration of individual nuclear medicine specialists, worldwide, in pioneering effective humane therapy of particular advanced cancers unresponsive to conventional treatments.


Assuntos
Neoplasias , Compostos Radiofarmacêuticos , Humanos , Ensaios de Uso Compassivo , Neoplasias/terapia , Medicina Nuclear/métodos , Medicina de Precisão/métodos , Compostos Radiofarmacêuticos/uso terapêutico
15.
Eur J Cancer ; 201: 113911, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38377774

RESUMO

BACKGROUND: Sotorasib is a first-in-class KRAS p.G12C-inhibitor that has entered clinical trials in pretreated patients with non-small cell lung cancer (NSCLC) in 2018. First response rates were promising in the CodeBreaK trials. It remains unclear whether response to sotorasib and outcomes differ in a real-world setting when including patients underrepresented in clinical trials. METHODS: Patients with KRAS p.G12C-mutated advanced or metastatic NSCLC received sotorasib within the German multicenter sotorasib compassionate use program between 2020 to 2022. Data on efficacy, tolerability, and survival were analyzed in the full cohort and in subgroups of special interest such as co-occurring mutations and across PD-L1 expression levels. RESULTS: We analyzed 163 patients who received sotorasib after a median of two treatment lines (range, 0 to 7). Every fourth patient had a poor performance status and 38% had brain metastases (BM). The objective response rate was 38.7%. The median overall survival was 9.8 months (95% CI, 6.5 to not reached). Median real-world (rw) progression-free survival was 4.8 months (9% CI, 3.9 to 5.9). Dose reductions and permanent discontinuation were necessary in 35 (21.5%) and 7 (4.3%) patients, respectively. Efficacy seems to be influenced by PD-L1 expression and a co-occurring KEAP1 mutation. KEAP1 was associated with an inferior survival. Other factors such as BM, STK11, and TP53 mutations had no impact on response and survival. CONCLUSION: First results from a real-world population confirm promising efficacy of sotorasib for the treatment of advanced KRAS p.G12C-mutated NSCLC. Patients with co-occurring KEAP1 mutations seem to derive less benefit.


Assuntos
Neoplasias Encefálicas , Carcinoma Pulmonar de Células não Pequenas , Neoplasias Pulmonares , Piperazinas , Piridinas , Pirimidinas , Humanos , Ensaios de Uso Compassivo , Antígeno B7-H1 , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Carcinoma Pulmonar de Células não Pequenas/genética , Proteína 1 Associada a ECH Semelhante a Kelch/genética , Proteínas Proto-Oncogênicas p21(ras)/genética , Neoplasias Pulmonares/tratamento farmacológico , Neoplasias Pulmonares/genética , Fator 2 Relacionado a NF-E2 , Alemanha , Mutação
17.
Biomed Pharmacother ; 171: 116055, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-38171239

RESUMO

BACKGROUND: During the first wave of COVID-19, hydroxychloroquine (HCQ) was used off-label despite the absence of evidence documenting its clinical benefits. Since then, a meta-analysis of randomised trials showed that HCQ use was associated with an 11% increase in the mortality rate. We aimed to estimate the number of HCQ-related deaths worldwide. METHODS AND FINDINGS: We estimated the worldwide in-hospital mortality attributable to HCQ use by combining the mortality rate, HCQ exposure, number of hospitalised patients, and the increased relative risk of death with HCQ. The mortality rate in hospitalised patients for each country was calculated using pooled prevalence estimated by a meta-analysis of published cohorts. The HCQ exposure was estimated using median and extreme estimates from the same systematic review. The number of hospitalised patients during the first wave was extracted from dedicated databases. The systematic review included 44 cohort studies (Belgium: k = 1, France: k = 2, Italy: k = 12, Spain: k = 6, Turkey: k = 3, USA: k = 20). HCQ prescription rates varied greatly from one country to another (range 16-84%). Overall, using median estimates of HCQ use in each country, we estimated that 16,990 HCQ-related in-hospital deaths (range 6267-19256) occurred in the countries with available data. The median number of HCQ-related deaths in Belgium, Turkey, France, Italy, Spain, and the USA was 240 (range not estimable), 95 (range 92-128), 199 (range not estimable), 1822 (range 1170-2063), 1895 (range 1475-2094) and 12739 (3244- 15570), respectively. CONCLUSIONS: Although our estimates are limited by their imprecision, these findings illustrate the hazard of drug repurposing with low-level evidence.


Assuntos
COVID-19 , Hidroxicloroquina , Humanos , Ensaios de Uso Compassivo , Tratamento Farmacológico da COVID-19 , Resultado do Tratamento
18.
Med Law Rev ; 32(1): 20-41, 2024 Feb 26.
Artigo em Inglês | MEDLINE | ID: mdl-37616571

RESUMO

Through the New Drugs and Clinical Trials Rules, 2019 (2019 Rules), India has developed the rules governing post-trial access (PTA) to new drugs or investigational new drugs. However, inconsistencies and interpretational challenges exist in the application of the 2019 Rules and the Indian Council of Medical Research Guidelines 2017. This conflation poses a real harm to the trial participants, specifically the ones with limited access to healthcare facilities. Since drug laws in India do not expressly deal with other forms of access like the 'Compassionate Use' or 'Expanded Access' mechanism, demarcating the scope and describing the strategies for PTA are the need of the hour. We propose possible strategies to address inadequacies in the regulatory regime and establish 'win-win' situations among all stakeholders. We further argue that India is well positioned to provide leadership by developing detailed PTA provisions and may set a potential path for the other clinical trial host countries.


Assuntos
Ensaios de Uso Compassivo , Drogas em Investigação , Humanos , Drogas em Investigação/uso terapêutico , Índia
20.
Pediatr Nephrol ; 39(3): 911-914, 2024 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-38086983

RESUMO

BACKGROUND: Erythropoiesis-stimulating agents (ESAs) have played an important role in the treatment of renal anemia in children, but cannot improve hemoglobin to target level in some cases. Roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, can stimulate endogenous erythropoietin production and regulate iron metabolism even in patients with kidney failure. However, roxadustat has not yet been approved for use in children. CASE-DIAGNOSIS/TREATMENT: We report a case of refractory renal anemia in an 80-day-old boy, who was hyporesponsive to ESAs even in combination with iron supplementation and transfusion. Compassionate use of roxadustat successfully corrected the intractable anemia. Hyperkalemia is a manageable adverse event of concern during follow-up. CONCLUSION: The successful experience in this case may inform the clinical utility of roxadustat for refractory renal anemia in children, which should be further confirmed by well-designed prospective clinical trials.


Assuntos
Anemia , Hematínicos , Insuficiência Renal Crônica , Masculino , Criança , Humanos , Ensaios de Uso Compassivo , Estudos Prospectivos , Insuficiência Renal Crônica/terapia , Anemia/etiologia , Anemia/induzido quimicamente , Hematínicos/efeitos adversos , Doença Crônica , Glicina/uso terapêutico , Glicina/farmacologia , Isoquinolinas/efeitos adversos , Ferro/uso terapêutico
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