Nouvelles thérapeutiques dans le champ des maladies neuromusculaires : quels enjeux ?

Bonnichon, Delphine; Bénézit, A.

doi:10.3917/eres.gargi.2020.01.0157

Cited by 2 publications

(4 citation statements)

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“…(10) "According to him, our son was not particularly ill, and it also did not seem to be a question of him not receiving treatment […] Anyway, I think that if we had absolutely wanted him not to be treated, I don't think the doctor would have necessarily objected. But to him, our son was a really good candidate for innovative therapy" (13).…”

Section: Qualitative Data From Semi-structured Interviewsmentioning

confidence: 99%

“…"In general, I think that the, well, I think we can talk a little about collaboration between the medical team and ourselves because, yeah, we are involved, and they are involved as well. She is really very, very good, we are really…Well, we realize how lucky we are to have this doctor and this whole medical team" (13).…”

Section: Theme 3: Whose Decision Is It? a Shared Decision Between Par...mentioning

confidence: 99%

“…He's been in really great shape since December and that's been really good for us." (13) "Right now I have a four-year-old girl, not one with spinal muscular atrophy, but a four-year-old girl with a motor delay" (14…”

Section: Theme 5: the Experience Of Decision-making In The Medium Ter...mentioning

confidence: 99%

“…When the MCM proposes innovative therapy, the final decision about whether to treat the child is always up to the parents. They may sometimes interpret this proposal as a solution repairing the shock of the announcement of the child's illness, particularly when the SMA is severe ( 13 ). After the final decision, therapies are most of the time initiated 2 weeks after the genetic diagnosis has been confirmed.…”

Section: Introductionmentioning

confidence: 99%

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Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

et al. 2022

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BackgroundSMA type 1 is a severe neurodegenerative disorder that, in the absence of curative treatment, leads to death before 1 year of age without ventilatory support. Three innovative therapies are available to increase life expectancy.Purpose(i) To increase knowledge about parents' experiences with their decision to have opted for an innovative therapy; (ii) to assess the middle-term psychological consequences in the parents' lives.MethodsWe used an in-depth interview; a self-administrated questionnaire and self-report scales (BDI-II, STAI-Y, PSI-SF, SOC-13, PBA, DAS 16 and FICD). We compared parents hesitant before the decision to parents who were not-hesitant and the group of parents whose child was treated with gene therapy (GT) to parents whose child received another innovative therapy.Main resultsWe included n = 18 parents of 13 children. Parent's mean age was 34.7 (±5.2), child's average age was 44.3 months (±38.0). Retrospectively, most parents felt involved by doctors in decision-making on treatment, they felt their point of view was considered and were satisfied with the effects of the treatment. The group of parents “non-hesitant” was more depressed (p < 0.001), more anxious (p = 0.022) and had higher parental stress (p = 0.026) than the group of “hesitant” parents; the group of “GT-treated” parents was more depressed (p = 0.036) than the group of parents with “other therapy”. Qualitative data highlights revealed: the need to save the child's life at all costs; the fear of coping with end of life and palliative care, the high value of perceived physician confidence in the treatment, the hope that the child will acquire autonomy or be cured. At the time of the decision, no parents felt they fully understood all of the issues regarding therapy and the disease.ConclusionHesitating before making a decision did not predispose parents to depression and anxiety. The narratives suggest that the parents faced a dilemma regarding their child's health in an urgent context. The decision was not final, and parents will continue to think about it throughout the care process.

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Section: Qualitative Data From Semi-structured Interviewsmentioning

confidence: 99%

Section: Theme 3: Whose Decision Is It? a Shared Decision Between Par...mentioning

confidence: 99%

Section: Theme 5: the Experience Of Decision-making In The Medium Ter...mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

et al. 2022

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Être parent d’un enfant porteur d’une amyotrophie spinale (SMA) à l’ère des nouvelles thérapies

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Nouvelles thérapeutiques dans le champ des maladies neuromusculaires : quels enjeux ?

Cited by 2 publications

References 4 publications

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

Être parent d’un enfant porteur d’une amyotrophie spinale (SMA) à l’ère des nouvelles thérapies

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Nouvelles thérapeutiques dans le champ des maladies neuromusculaires : quels enjeux ?

Cited by 2 publications

References 4 publications

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

Être parent d’un enfant porteur d’une amyotrophie spinale (SMA) à l’ère des nouvelles thérapies

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Nouvelles thérapeutiques dans le champ des maladies neuromusculaires : quels enjeux ?